Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period.
LAS VEGAS, June 5, 2025 /PRNewswire/ — DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Friedreich's Ataxia Market Report
According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034.
The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals.
Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing.
Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years.
The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others.
Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report
Friedreich's Ataxia Market Dynamics
The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology.
The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification.
Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM.
However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies.
Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth.
Friedreich's Ataxia Treatment Market
Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected.
The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective.
Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles.
Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation.
SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects.
It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone.
To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication
Friedreich's Ataxia Pipeline Therapies and Key Companies
Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others.
Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025.
RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021.
Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition.
The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials
Recent Developments in the Friedreich's Ataxia Market
In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression.
In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review.
In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia.
Friedreich's Ataxia Overview
Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination.
The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease.
In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech.
Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis.
Friedreich's Ataxia Epidemiology Segmentation
The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Diagnosed Prevalent Cases of Friedreich's Ataxia
Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia
Total Treated Cases of Friedreich's Ataxia
Friedreich's Ataxia Market Report Metrics
Details
Study Period
2020–2034
Coverage
7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan].
Key Friedreich's Ataxia Companies
Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, Reata Pharmaceuticals, and others
Key Friedreich's Ataxia Therapies
RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, SKYCLARYS, and others
Scope of the Friedreich's Ataxia Market Report
Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies
Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook
Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement
Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market
Table of Contents
1.
Friedreich's Ataxia Market Key Insights
2.
Friedreich's Ataxia Market Report Introduction
3.
Friedreich's Ataxia Market Overview at a Glance
4.
Friedreich's Ataxia Market Executive Summary
5.
Disease Background and Overview
6.
Friedreich's Ataxia Treatment and Management
7.
Friedreich's Ataxia Epidemiology and Patient Population
8.
Patient Journey
9.
Friedreich's Ataxia Marketed Drugs
10.
Friedreich's Ataxia Emerging Drugs
11.
Seven Major Friedreich's Ataxia Market Analysis
12.
Friedreich's Ataxia Market Outlook
13.
Potential of Current and Emerging Therapies
14.
KOL Views
15.
Unmet Needs
16.
SWOT Analysis
17.
Appendix
18.
DelveInsight Capabilities
19.
Disclaimer
20.
About DelveInsight
Related Reports
Friedreich's Ataxia Epidemiology Forecast
Friedreich's Ataxia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Friedreich's ataxia epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Friedreich's Ataxia Pipeline
Friedreich's Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Friedreich's ataxia companies, including PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, among others.
Ataxia Market
Ataxia Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key ataxia companies, including AstraZeneca, Merck Sharp & Dohme LLC, Bayer, Roche, among others.
Ataxia Pipeline
Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ataxia companies, including Retrotope, Reata Pharmaceuticals, PTC Therapeutics, Metro International Biotech, LLC, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, EryDel, Biogen, Matrix Biomed, IntraBio, Biohaven Pharmaceuticals, Inc., Stealth BioTherapeutics Inc., Acasti Pharma, Seelos Therapeutics, Kissei Pharmaceutical, Vico Therapeutics, Q-State Biosciences, Locanabio, Lexeo Therapeutics, Voyager Therapeutics, CRISPR Therapeutics, Capsida Biotherapeutics, AavantiBio, StrideBio, Wave Life Sciences, REPROCELL, SHIONOGI & Co., CORESTEM, Blade Therapeutics, Exicure, Lacerta Therapeutics, Healx Ltd., Uniqure, Ionis Pharmaceuticals, Jupiter Neurosciences, among others.
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.
Contact UsShruti Thakur [email protected] +14699457679www.delveinsight.com
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Delta-like Ligand 3 Targeted Therapies Market is Predicted to Heat Up During the Forecast Period (2025-2034) Across 7MM Owing to a Robust Pipeline

The delta-like ligand 3 targeted therapies market is witnessing significant growth, driven by the rising incidence of small cell lung cancer (SCLC) and neuroendocrine tumors. Advancements in antibody-drug conjugate (ADC) technology and increasing clinical trial activity are propelling the development of DLL3-targeted candidates. Growing interest from biopharma companies and strategic collaborations are also accelerating the DLL3-targeted therapies market expansion. LAS VEGAS, June 19, 2025 /PRNewswire/ — DelveInsight's Delta-like Ligand 3 Targeted Therapies Market Size, Target Population, Competitive Landscape & Market Forecast report includes a comprehensive understanding of current treatment practices, emerging delta-like ligand 3 targeted therapies, market share of individual therapies, and current and forecasted delta-like ligand 3 targeted therapies market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Delta-like Ligand 3 Targeted Therapies Market Report As per DelveInsight's analysis, the total market size of delta-like ligand 3 targeted therapies in the 7MM is expected to surge significantly by 2034. The approval of the first DLL3-targeted Bispecific T-cell Engager therapy, IMDELLTRA, by the US Food and Drug Administration (FDA) in May 2024 marked a pivotal moment for patients battling Extensive-stage small-cell lung Cancer (ES-SCLC). Leading delta-like ligand 3 targeted therapies companies, such as Phanes Therapeutics, Merck, Daiichi Sankyo, Legend Biotech, Novartis, Abdera Therapeutics, Boehringer Ingelheim, Chugai Pharmaceutical, Roche, Molecular Partners, Orano Med, Zai Lab, Allogene Therapeutics, and others, are developing novel delta-like ligand 3 targeted therapies that can be available in the delta-like ligand 3 targeted therapies market in the coming years. Some of the key delta-like ligand 3 targeted therapies in the pipeline include Peluntamig (PT217), MK-6070/DS3280, LB2102/ DLL3-targeted CAR Ts, 225Ac-ABD147, Obrixtamig (BI 764532), ALPS12/RG6524, MP0712, ZL-1310, ALL-213, and others. In February 2025, Phanes Therapeutics announced that the first patient was dosed in the clinical study of peluntamig (PT217) in combination with chemotherapy. In September 2024, Abdera Therapeutics announced that the US FDA granted ODD to ABD-147 for the treatment of NEC. In August 2024, Daiichi Sankyo and Merck expanded their existing global co-development and co-commercialization agreement for Merck's MK-6070 for the treatment of SCLC. Discover which indication is expected to grab the major delta-like ligand 3 targeted therapies market share @ Delta-like Ligand 3 Targeted Therapies Market Report Delta-like Ligand 3 Targeted Therapies Market Dynamics The delta-like ligand 3 targeted therapies market is emerging as a dynamic and promising area in oncology, particularly for hard-to-treat cancers like SCLC and other neuroendocrine tumors. With a growing understanding of its role in tumorigenesis, drug developers have shifted focus toward exploiting this target through various modalities, including ADCs, BiTEs, and CAR-T cell therapies. The FDA approval of tarlatamab (IMDELLTRA), a DLL3-targeted BiTE, has catalyzed commercial and clinical interest in this space. The competitive landscape is rapidly evolving, driven by both established pharmaceutical players and biotech innovators. Several companies are advancing DLL3-targeted assets through clinical pipelines, highlighting the growing investment and belief in the target's therapeutic value. Tarlatamab, in particular, has set a precedent by demonstrating durable responses in patients with extensive-stage SCLC who have limited treatment options after frontline chemotherapy. Its clinical success has validated DLL3 as a viable target and opened the door for additional programs in earlier lines of therapy and combination regimens. 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Reimbursement strategies and payer acceptance will further impact uptake, especially given the premium pricing associated with novel biologics. Looking ahead, the DLL3-targeted therapies market is poised for expansion beyond SCLC. Preliminary studies are exploring DLL3 expression in other neuroendocrine carcinomas, prostate cancer, and some pediatric tumors. This potential for label expansion, coupled with advancing drug formats and supportive regulatory pathways, indicates a robust growth trajectory. As the clinical pipeline matures and real-world data accumulate, DLL3-targeted approaches are expected to play an increasingly integral role in the precision oncology landscape. Delta-like Ligand 3 Targeted Therapies Treatment Market DLL3-targeted therapies are emerging as a promising new approach in the treatment of small-cell lung cancer and neuroendocrine carcinomas, bringing renewed optimism for patients with these aggressive cancers. 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In August 2024, Daiichi Sankyo and Merck expanded their global partnership to include MK-6070 under their co-development and co-commercialization agreement for DXd antibody-drug conjugates. Merck retains exclusive rights in Japan and will handle all manufacturing and supply responsibilities for MK-6070. The two companies also plan to explore the use of MK-6070 in combination with ifinatamab deruxtecan (I-DXd) for SCLC, along with other potential combination strategies. The anticipated launch of these emerging therapies is poised to transform the delta-like ligand 3 targeted therapies market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the delta-like ligand 3 targeted therapies market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about delta-like ligand 3 targeted therapies clinical trials, visit @ Delta-like Ligand 3 Targeted Therapies Treatment Delta-like Ligand 3 Targeted Therapies Overview Delta-like ligand 3 (DLL3) targeted therapies is an unconventional ligand within the Notch signaling pathway that, when overexpressed, supports the growth, migration, and invasiveness of small cell lung cancer (SCLC) cells. It also contributes to the development of metastatic and therapy-resistant traits in neuroendocrine carcinomas (NECs), enhancing both tumor cell proliferation and resistance to platinum-based chemotherapy. In normal cells, DLL3 expression is minimal and confined to the Golgi apparatus and cytoplasmic vesicles. This specific intracellular localization is regulated by its transmembrane domain and nearby protein sequences, which serve as retention signals. In contrast, SCLC cells exhibit high levels of DLL3 that are abnormally localized to the cell surface, a characteristic observed in up to 85% of human SCLC cases. While the mechanisms behind this overexpression and altered trafficking are not fully understood, DLL3's distinct expression pattern positions it as a promising biomarker and therapeutic target in SCLC. Beyond SCLC, DLL3 is also widely expressed in various NECs, including certain molecular subtypes of pulmonary large cell NEC (LCNEC) and NECs originating from the gastroenteropancreatic tract, bladder, prostate, and cervix. In these tumors, elevated DLL3 expression correlates with more advanced disease and poorer overall survival, suggesting a strong association between high DLL3 levels and unfavorable clinical outcomes. Delta-like Ligand 3 Targeted Therapies Epidemiology Segmentation The delta-like ligand 3 targeted therapies market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Cases in Selected Indications for DLL3-targeted Therapies Total Eligible Patient Pool in Selected Indications for DLL3-targeted Therapies Total Treated Cases in Selected Indications for DLL3-targeted Therapies Delta-like Ligand 3 Targeted Therapies Report Metrics Details Study Period 2020–2034 Delta-like Ligand 3 Targeted Therapies Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] Key Delta-like Ligand 3 Targeted Therapies Companies Phanes Therapeutics, Merck, Daiichi Sankyo, Legend Biotech, Novartis, Abdera Therapeutics, Boehringer Ingelheim, Chugai Pharmaceutical, Roche, Molecular Partners, Orano Med, Zai Lab, Allogene Therapeutics, Amgen, and others Key Delta-like Ligand 3 Targeted Therapies Peluntamig (PT217), MK-6070/DS3280, LB2102/ DLL3-targeted CAR Ts, 225Ac-ABD147, Obrixtamig (BI 764532), ALPS12/RG6524, MP0712, ZL-1310, ALL-213, IMDELLTRA, and others Scope of the Delta-like Ligand 3 Targeted Therapies Market Report Delta-like Ligand 3 Targeted Therapies Therapeutic Assessment: Delta-like Ligand 3 Targeted Therapies current marketed and emerging therapies Delta-like Ligand 3 Targeted Therapies Market Dynamics: Conjoint Analysis of Emerging Delta-like Ligand 3 Targeted Therapies Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Delta-like Ligand 3 Targeted Therapies Market Access and Reimbursement Discover more about delta-like ligand 3 targeted therapies in development @ Delta-like Ligand 3 Targeted Therapies Clinical Trials Table of Contents 1. Key Insights 2. Report Introduction 3. Executive Summary 4. Key Events 5. Market Forecast Methodology 6. Delta-like Ligand 3 Targeted Therapies Market Overview at a Glance in the 7MM 6.1. Market Share (%) Distribution by Therapies in 2025 6.2. Market Share (%) Distribution by Therapies in 2034 6.3. Market Share (%) Distribution by Indications in 2025 6.4. Market Share (%) Distribution by Indications in 2034 7. Delta-like Ligand 3 (DLL3)-targeted Therapies: Background and Overview 7.1. Introduction 7.2. The Potential of Delta-like Ligand 3 (DLL3)-targeted Therapies in Different Indications 7.3. Clinical Applications of Delta-like Ligand 3 (DLL3)-targeted Therapies 8. Target Patient Pool of Delta-like Ligand 3 (DLL3)-targeted Therapies 8.1. Assumptions and Rationale 8.2. Key Findings 8.3. Total Cases of Selected Indication for Delta-like Ligand 3 (DLL3)-targeted Therapies in the 7MM 8.4. Total Eligible Patient Pool of Selected Indication for Delta-like Ligand 3 (DLL3)-targeted Therapies in the 7MM 8.5. Total Treatable Cases in Selected Indication for Delta-like Ligand 3 (DLL3)-targeted Therapies in the 7MM 9. Marketed Therapies 9.1. Key Competitors 9.2. IMDELLTRA (tarlatamab-dlle): Amgen 9.2.1. Product Description 9.2.2. Regulatory milestones 9.2.3. Other developmental activities 9.2.4. Clinical development 9.2.5. Safety and efficacy List to be continued in the report 10. Emerging Therapies 10.1. Key Competitors 10.2. Peluntamig (PT217): Phanes Therapeutics 10.2.1. Product Description 10.2.2. Other developmental activities 10.2.3. Clinical development 10.2.4. Safety and efficacy 10.3. MK-6070/ HPN328-4001/ DS3280: Merck/ Daiichi Sankyo 10.3.1. Product Description 10.3.2. Other developmental activities 10.3.3. Clinical development 10.3.4. Safety and efficacy List to be continued in the report 11. Delta-like Ligand 3 Targeted Therapies: Seven Major Market Analysis 11.1. Key Findings 11.2. Market Outlook 11.3. Conjoint Analysis 11.4. Key Market Forecast Assumptions 11.4.1. Cost Assumptions and Rebates 11.4.2. Pricing Trends 11.4.3. Analogue Assessment 11.4.4. Launch Year and Therapy Uptakes 11.5. Total Market Sizes of Delta-like Ligand 3 (DLL3)-targeted Therapies by Indications in the 7MM 11.6. The United States Market Size 11.6.1. Total Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies in the United States 11.6.2. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Indication in the United States 11.6.3. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Therapies in the United States 11.7. EU4 and the UK 11.7.1. Total Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies in EU4 and the UK 11.7.2. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Indications in EU4 and the UK 11.7.3. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Therapies in EU4 and the UK 11.8. Japan 11.8.1. Total Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies Inhibitors in Japan 11.8.2. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Indications in Japan 11.8.3. Market Size of Delta-like Ligand 3 (DLL3)-targeted Therapies by Therapies in Japan 12. SWOT Analysis 13. KOL Views 14. Unmet Needs 15. Market Access and Reimbursement 16. Appendix 16.1. Bibliography 16.2. Report Methodology 17. DelveInsight Capabilities 18. Disclaimer 19. About DelveInsight Related Reports Small Cell Lung Cancer Market Small Cell Lung Cancer Disorder Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SCLC companies, including Ascentage Pharma, Merck & Co, AstraZeneca, Advenchen Laboratories, GlaxoSmithKline, Advanced Accelerator Applications, Trillium Therapeutics, Vernalis, Oncoceutics, NewBio Therapeutics, Wigen Biomedicine, Linton Pharm, Carrick Therapeutics, Xencor, Jiangsu HengRui Medicine, Aileron Therapeutics, Roche, Ipsen, Celgene, Lee's Pharmaceutical Limited, AbbVie, G1 Therapeutics, Chipscreen Biosciences, Luye Pharma Group, Shanghai Henlius Biotech, CSPC ZhongQi Pharmaceutical Technology, Impact Therapeutics, among others. 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About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content:

Malaysian Reserve

05-06-2025

• Malaysian Reserve

Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period. LAS VEGAS, June 5, 2025 /PRNewswire/ — DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Friedreich's Ataxia Market Report According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034. The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals. Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing. Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years. The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others. Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report Friedreich's Ataxia Market Dynamics The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology. The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification. Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM. However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies. Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth. Friedreich's Ataxia Treatment Market Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected. The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective. Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles. Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation. SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects. It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone. To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication Friedreich's Ataxia Pipeline Therapies and Key Companies Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others. Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025. RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021. Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition. The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials Recent Developments in the Friedreich's Ataxia Market In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression. In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review. In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia. Friedreich's Ataxia Overview Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination. The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease. In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech. Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis. Friedreich's Ataxia Epidemiology Segmentation The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalent Cases of Friedreich's Ataxia Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia Total Treated Cases of Friedreich's Ataxia Friedreich's Ataxia Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan]. Key Friedreich's Ataxia Companies Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, Reata Pharmaceuticals, and others Key Friedreich's Ataxia Therapies RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, SKYCLARYS, and others Scope of the Friedreich's Ataxia Market Report Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market Table of Contents 1. Friedreich's Ataxia Market Key Insights 2. Friedreich's Ataxia Market Report Introduction 3. Friedreich's Ataxia Market Overview at a Glance 4. Friedreich's Ataxia Market Executive Summary 5. Disease Background and Overview 6. Friedreich's Ataxia Treatment and Management 7. Friedreich's Ataxia Epidemiology and Patient Population 8. Patient Journey 9. Friedreich's Ataxia Marketed Drugs 10. Friedreich's Ataxia Emerging Drugs 11. Seven Major Friedreich's Ataxia Market Analysis 12. Friedreich's Ataxia Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis 17. Appendix 18. DelveInsight Capabilities 19. Disclaimer 20. About DelveInsight Related Reports Friedreich's Ataxia Epidemiology Forecast Friedreich's Ataxia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Friedreich's ataxia epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Friedreich's Ataxia Pipeline Friedreich's Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Friedreich's ataxia companies, including PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, among others. Ataxia Market Ataxia Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key ataxia companies, including AstraZeneca, Merck Sharp & Dohme LLC, Bayer, Roche, among others. Ataxia Pipeline Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ataxia companies, including Retrotope, Reata Pharmaceuticals, PTC Therapeutics, Metro International Biotech, LLC, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, EryDel, Biogen, Matrix Biomed, IntraBio, Biohaven Pharmaceuticals, Inc., Stealth BioTherapeutics Inc., Acasti Pharma, Seelos Therapeutics, Kissei Pharmaceutical, Vico Therapeutics, Q-State Biosciences, Locanabio, Lexeo Therapeutics, Voyager Therapeutics, CRISPR Therapeutics, Capsida Biotherapeutics, AavantiBio, StrideBio, Wave Life Sciences, REPROCELL, SHIONOGI & Co., CORESTEM, Blade Therapeutics, Exicure, Lacerta Therapeutics, Healx Ltd., Uniqure, Ionis Pharmaceuticals, Jupiter Neurosciences, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur [email protected] + Logo: View original content:

Malaysian Reserve

20-05-2025

• Malaysian Reserve

MAC Lung Disease Market Poised for Growth Across the 7MM During the Forecast Period (2025-2034) Amid Rising Prevalence and New Therapeutic Solutions

The MAC lung disease market is predicted to grow in the coming years. This growth is mainly driven by the launch of innovative therapies such as MNKD-101, Bedaquiline fumarate (TMC207), and SPR720, among others. Furthermore, the increasing prevalence of MAC lung disease, influenced by host factors like advanced age, COPD, thoracic abnormalities, and environmental exposure to MAC in soil, water, and aerosol-generating activities, is expected to expand the MAC lung disease market potential. LAS VEGAS, May 20, 2025 /PRNewswire/ — Mycobacterium avium Complex (MAC) refers to a group of bacteria, primarily Mycobacterium avium and Mycobacterium intracellulare, that are responsible for MAC lung disease. Accurate identification typically requires genetic testing. MAC is the leading cause of nontuberculous mycobacterial (NTM) lung infections in the United States and can affect people of all ages, although it is more common in older adults, postmenopausal women, smokers, and individuals with weakened immune systems or pre-existing lung conditions such as bronchiectasis, COPD, cystic fibrosis, emphysema, or a prior history of tuberculosis. DelveInsight estimates that in 2023, there were around 119K diagnosed prevalent cases of MAC lung disease across the 7MM, with the United States accounting for approximately 64% of these cases. In 2023, there were an estimated 151K diagnosed prevalent cases of NTM lung disease across the 7MM, with the number expected to rise by 2034. Treatment for MAC lung disease typically involves a combination of multiple antibiotics, most often including a macrolide (such as azithromycin or clarithromycin), a rifamycin (such as rifampin or rifabutin), and ethambutol. This regimen works by inhibiting protein synthesis, disrupting the bacterial cell wall, and preventing replication of the mycobacteria. Therapy generally continues for 12 to 18 months, depending on the severity of the disease and the pathogen's drug susceptibility. Supportive therapies play an important role, especially for patients with preexisting lung conditions. Chest physiotherapy, mucolytic agents, and bronchodilators help clear mucus and alleviate airway obstruction. Additionally, devices like positive expiratory pressure (PEP) masks and high-frequency chest wall oscillation (HFCWO) devices can assist in reducing bacterial burden. Learn more about the MAC lung disease treatment @ New Treatment for MAC Lung Disease ARIKAYCE is the first FDA-approved therapy specifically for MAC lung disease. It is a once-daily inhaled formulation of amikacin, delivered using Insmed's proprietary PULMOVANCE technology to directly target the lungs while reducing systemic exposure. The treatment is administered through the Lamira Nebulizer System and was granted orphan drug status by the FDA in 2013. In May 2024, new data from the ARISE study were presented at ATS 2024, highlighting outcomes in MAC lung disease patients who had not previously received antibiotic therapy. ARIKAYCE is approved in the US, European Union, and Japan, and is currently being studied in the Phase III ENCORE trial, which focuses on newly diagnosed or relapsed MAC patients who have not begun antibiotic treatment. Topline results from this study are anticipated in the first quarter of 2026. Find out more on FDA-approved MAC lung disease drugs @ MAC Lung Disease Treatment Options The scarcity of FDA-approved therapies for MAC lung disease, with only ARIKAYCE being the only approved option, limits treatment options, complicates management of resistant strains, and highlights the need for new therapies. MannKind Corporation, Janssen Pharmaceutical, and Spero Therapeutics are progressing through various stages of clinical trials, driving innovation in the MAC lung disease market. This activity is creating a dynamic environment, offering significant opportunities for the MAC lung disease market expansion and growth. Among the emerging therapies, MNKD-101, a nebulized formulation of clofazimine, is currently in Phase III clinical development and is expected to launch in the market by 2027. Discover which therapies are expected to grab major MAC lung disease market share @ MAC Lung Disease Market Report MNKD-101 is a nebulized formulation of clofazimine being developed to treat severe, chronic, and recurrent lung infections, such as nontuberculous mycobacterial lung disease. This inhaled version is anticipated to provide therapeutic benefits over the existing oral form. MannKind is also working on a dry-powder variant using its proprietary Technosphere platform. In May 2024, the U.S. FDA granted Fast Track Designation to MNKD-101 for NTM lung disease, following its earlier designations as an orphan drug and a Qualified Infectious Disease Product (QIDP). The FDA approved the Investigational New Drug (IND) application for MNKD-101 in April 2024, paving the way for a Phase III clinical trial. Similarly, Japan's PMDA authorized the Phase III ICoN-1 trial in September 2024, enabling the global trial to move forward. Bedaquiline fumarate, marketed as SIRTURO, is a diarylquinoline antimycobacterial agent used in combination regimens to treat pulmonary tuberculosis caused by Mycobacterium tuberculosis strains resistant to both rifampin and isoniazid. It is approved for use in adults and children aged 5 years and older, weighing at least 15 kg. Bedaquiline is currently being studied in a Phase II/III clinical trial as part of a treatment regimen with clarithromycin and ethambutol for adults with treatment-refractory Mycobacterium avium complex lung disease (MAC-LD). Spero Therapeutics is developing SPR720, an oral therapy for NTM pulmonary disease. SPR720 is a stable prodrug that is rapidly converted into SPR719, which targets the ATPase subunits of gyrase and topoisomerase, mechanisms distinct from fluoroquinolones. Preclinical data have demonstrated SPR720's broad-spectrum activity against key NTM pathogens such as MAC, M. kansasii, and M. abscessus, in both treatment-refractory and treatment-naïve patients. The FDA has granted SPR720 Fast Track status, orphan drug designation, and QIDP designation. Discover more about drugs for MAC lung disease in development @ MAC Lung Disease Clinical Trials The anticipated launch of these emerging therapies for MAC lung disease are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the MAC lung disease market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for MAC lung disease is expected to grow from USD 474 million in the 7MM in 2023 at a significant CAGR by 2034. The market growth across the 7MM is expected to be fueled by the introduction of novel treatments such as MNKD-101, Bedaquiline fumarate (TMC207), and SPR720, among others. Additionally, the rising incidence of MAC lung disease driven by factors like aging populations, COPD, structural lung abnormalities, and exposure to MAC through soil, water, and aerosol-generating activities will likely contribute to an expanding MAC lung disease market opportunity. DelveInsight's latest published market report, titled as MAC Lung Disease Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the MAC lung disease country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The MAC lung disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of NTM Lung Disease Gender-specific Diagnosed Prevalent Cases of NTM Lung Disease Species-specific Diagnosed Prevalent Cases of NTM Infection Total Diagnosed Prevalent Cases of MAC Lung Disease The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM MAC lung disease market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this MAC lung disease market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the MAC lung disease market. Also, stay abreast of the mitigating factors to improve your market position in the MAC lung disease therapeutic space. Related Reports Nontuberculous Mycobacterial Infection Market Nontuberculous Mycobacterial Infection Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key NTM infections companies, including AN2 Therapeutics, Mannkind Corporation, Spero Therapeutics, Paratek Pharmaceuticals, Redhill Biopharma, among others. Nontuberculous Mycobacterial Infection Pipeline Nontuberculous Mycobacterial Infection Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nontuberculous mycobacterial infection companies, including MannKind Corporation, Matinas BioPharma, Spero Therapeutics, Crestone, Inc., Vast Therapeutics, among others. Chronic Obstructive Pulmonary Disease Market Chronic Obstructive Pulmonary Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key chronic obstructive pulmonary disease companies, including Genentech, Inc., GlaxoSmithKline, Verona Pharma plc, Regeneron Pharmaceuticals, Sanofi, MedImmune LLC, EpiEndo Pharmaceuticals, Tetherex Pharmaceuticals, AstraZeneca, Chiesi Farmaceutici S.p.A., Synairgen Research Ltd., Mereo Biopharma, Organicell Regenerative Medicine, Pulmotect, Inc., Inmunotek S.L., PULMATRiX, GLENMARK PHARMACEUTICALS LTD, Dimerix Limited, ProterixBio, among others. Asthma Market Asthma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key asthma companies including GlaxoSmithKline, 4D Pharma plc, AstraZeneca, Suzhou Connect Biopharmaceuticals, Avillion, Pearl Therapeutics, ARS Pharmaceuticals, Sinomab, Avalo Therapeutics, Kymab, Sanofi, Cumberland Pharmaceuticals, Genentech, Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content: