Latest news with #NewDrugApplication
Business Wire
8 hours ago
- Business
- Business Wire
Arrowhead Pharmaceuticals Completes Enrollment in SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 Studies of Plozasiran
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has completed enrollment in SHASTA-3, SHASTA-4, and MUIR-3, the company's global Phase 3 clinical studies designed to support regulatory submissions for marketing approval of investigational plozasiran in the treatment of severe hypertriglyceridemia. Arrowhead anticipates completing the primary portion of these studies in mid-2026 with topline data expected shortly thereafter and planned submissions for regulatory review and potential approval to follow. The company previously submitted a New Drug Application (NDA) for plozasiran based on positive Phase 3 PALISADE study results in patients with familial chylomicronemia syndrome, which the U.S. FDA has accepted with a Prescription Drug User Fee Act (PDUFA) action date set for November 18, 2025. Arrowhead anticipates completing the primary portion of these studies in mid-2026 with topline data expected shortly thereafter and planned submissions for regulatory review and potential approval to follow. 'Completion of enrollment for the SHASTA-3, SHASTA-4, and MUIR-3 studies represent important milestones for plozasiran,' said Bruce Given, M.D., Chief Medical Scientist at Arrowhead. 'These studies bring us closer to generating the datasets needed to support potential global regulatory submissions for plozasiran as a treatment for severe hypertriglyceridemia, pending positive results. With a primary endpoint focused on triglyceride reduction at 12 months, we remain on track for study completion by mid-2026. Arrowhead's clinical development, clinical operations, and regulatory teams continue to execute complex global clinical studies with speed and precision. We owe the entire team involved a debt of gratitude for efficiently initiating these studies in 24 countries and enrolling approximately 2,200 patients. We're grateful to the investigators, caregivers, and patients for participating in these clinical studies, and we thank them for their trust in Arrowhead and in plozasiran.' About Severe Hypertriglyceridemia Severe hypertriglyceridemia (SHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL, with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL. SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Elevated TGs can also increase the risk of atherosclerotic cardiovascular disease (ASCVD). Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds. About SHASTA-3 and SHASTA-4 Phase 3 Studies SHASTA-3 (NCT06347003) and SHASTA-4 (NCT06347016) are double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of plozasiran in adults with severe hypertriglyceridemia. Between the two studies, approximately 750 participants were randomized to receive 4 doses (once every 3 months) of 25 mg plozasiran or placebo. The primary endpoint is percent change in fasting serum triglyceride levels from baseline to month 12 compared to placebo. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension. About MUIR-3 Phase 3 Study MUIR-3 (NCT06347133) is a double-blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of plozasiran in adults with hypertriglyceridemia (TGs greater than 150 mg/dL and less than 500 mg/dL). Approximately 1,450 participants were randomized to receive 4 doses (once every 3 months) of 25 mg plozasiran or placebo. The primary endpoint is percent change in fasting serum triglyceride levels from baseline to month 12 compared to placebo. About Plozasiran Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels. In multiple clinical studies, investigational plozasiran has demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. Plozasiran has been generally well tolerated to date with treatment emergent adverse events reported that generally reflect the comorbidities and underlying conditions of the study populations. Across clinical studies and study populations, the most frequently reported treatment emergent adverse events for the 25 mg dose that is proposed for marketing approval were COVID-19, upper respiratory tract infection, headache, Type 2 diabetes mellitus, and abdominal pain. Plozasiran is being investigated in the SUMMIT program of clinical studies, including the PALISADE Phase 3 study in patients with FCS, the SHASTA studies in patients with SHTG, and the MUIR studies in patients with mixed hyperlipidemia. Plozasiran in the treatment of patients with FCS has been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the U.S. Food and Drug Administration and Orphan Medicinal Product Designation by the European Medicines Agency. Investigational plozasiran has been submitted for marketing authorization in treatment of FCS to multiple global regulatory authorities but has not been reviewed or approved to treat any disease. About Arrowhead Pharmaceuticals, Inc. Arrowhead Pharmaceuticals, Inc. develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company's email list and receive news directly, please visit Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as 'may,' 'might,' 'will,' 'expect,' 'believe,' 'anticipate,' 'goal,' 'endeavor,' 'strive,' 'hope,' 'intend,' 'plan,' 'project,' 'could,' 'estimate,' 'potential,' 'target,' 'forecast' or 'continue' or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding regulatory approval for and commercial launch of plozasiran; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.
Yahoo
3 days ago
- Business
- Yahoo
Pfizer (NYSE:PFE) Joins FIBRE Consortium To Advance Fibrosis Treatment Through Innovative Imaging
The recent formation of the FIBRE Consortium, a collaboration involving Pfizer and other pharmaceutical partners, aims to pioneer advancements in fibrotic disease management. Alongside this, Pfizer's submission of a New Drug Application for vepdegestrant to treat breast cancer and significant progress in colorectal and prostate cancer therapies were key developments. Over the past month, Pfizer's stock saw a price increase of 4%. The company's performance appeared aligned with market trends amid geopolitical tensions and fluctuations in oil prices, indicating resilience within the broader mixed market conditions. These recent strategic initiatives have likely provided a positive foundation. We've identified 3 possible red flags with Pfizer and understanding the impact should be part of your investment process. Uncover 17 companies that survived and thrived after COVID and have the right ingredients to survive Trump's tariffs. The developments in Pfizer's strategic initiatives, specifically the formation of the FIBRE Consortium and the submission of the New Drug Application for vepdegestrant, could potentially enhance the company's revenue and earnings forecasts. These projects may strengthen Pfizer's position in the competitive pharmaceuticals landscape, especially as the company intensifies its focus on oncology. However, the ongoing threat of increased competition and reduced utilization of key drugs remain factors that may pressure revenue, possibly mitigating some of the positive impacts from the recent news. Over the past five years, Pfizer's total return, including share price and dividends, was a 1.05% decline, illustrating longer-term challenges despite short-term gains. On a one-year basis, the stock underperformed the US market, which achieved a 10.4% return, indicating some operational hurdles within the company. The recent strategic progress may offer potential relief if executed effectively. The stock's performance, with a recent monthly increase of 4%, places it in a unique position relative to the consensus analyst price target of US$29.24. The current share price of US$24.41 sits at a 22% discount to this target, hinting at potential upside according to more optimistic analysts. In contrast, some bearish analysts set a price target closer to US$24.57, suggesting that expectations are diverse. This disparity in views underscores the importance of closely monitoring how Pfizer's strategic developments influence its future performance and valuation. Jump into the full analysis health report here for a deeper understanding of Pfizer. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NYSE:PFE. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
6 days ago
- Business
- Business Wire
Bayer Files for Approval of gadoquatrane in the U.S.
WHIPPANY, N.J.--(BUSINESS WIRE)--Bayer today announced the submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its investigational contrast agent gadoquatrane for contrast-enhanced magnetic resonance imaging of the CNS and other body regions in adults and pediatric patients including term neonates. The submitted dose is 0.04 mmol gadolinium per kilogram body weight. If approved, gadoquatrane would become the lowest dose macrocyclic GBCA available in the U.S., corresponding to a 60 percent reduction compared to macrocyclic GBCAs dosed at 0.1 mmol Gd/kg body weight. The submission of gadoquatrane to the US FDA is based on positive data from the pivotal Phase III QUANTI studies evaluating the efficacy and safety of gadoquatrane in adult and pediatric patients globally. The first results from the Phase III QUANTI CNS study were presented at the European Congress of Radiology (ECR) in February of this year and further results are planned to be presented at upcoming scientific meetings. Nearly 40 million MRI scans are performed each year in the United States. 'There is a rising need for medical imaging, among others driven by the increasing incidence of chronic diseases such as cancer and cardiovascular diseases,' said Dr. Konstanze Diefenbach, Head of Radiology Research & Development, Bayer. 'As a leader in radiology, we are committed to advancing innovation in this field, including options to reduce the gadolinium dose. Patients can benefit from a dose reduction, especially patients with chronic conditions who require multiple contrast-enhanced MRI examinations during their lifetime. This is in line with guidance from health authorities and guidelines from scientific bodies which recommend using the lowest dose required to obtain the needed clinical information.' Bayer recently announced the submission of gadoquatrane to the Ministry of Health, Labour, and Welfare (MHLW) in Japan, marking the first application for marketing authorization for the investigational contrast agent. Further regulatory applications to health authorities worldwide are planned for the coming months. About the Phase III development program QUANTI The pivotal QUANTI clinical development program investigated gadoquatrane at a dose of 0.04 mmol Gd/kg body weight, which represents a 60 percent lower gadolinium dose compared to macrocyclic contrast agents dosed at 0.1 mmol Gd/kg body weight. QUANTI consisted of two large multinational, randomized, prospective double-blind, crossover Phase III studies – QUANTI CNS (Central Nervous System) and QUANTI OBR (Other Body Regions) – as well as the QUANTI Pediatric study. In total, 808 patients in 15 countries participated in the program. The QUANTI study results show that gadoquatrane met the primary and secondary efficacy endpoints of the studies assessing visualization parameters and lesion detection. Results of QUANTI Pediatric demonstrated that the pharmacokinetic behavior of gadoquatrane in children is similar to that in adults. The observed safety profile in adults as well as pediatric patients from birth to < 18 years of age in the QUANTI studies was generally consistent with previous data on gadoquatrane and other macrocyclic GBCAs. No new safety signals were observed. About gadoquatrane Gadoquatrane is Bayer's investigational extracellular macrocyclic contrast agent in clinical development for contrast enhancement in MRI. This low dose gadolinium-based contrast agent features a distinct tetrameric structure with high stability and high relaxivity. About MRI With an estimated 65 million procedures performed annually worldwide, contrast-enhanced MRI plays a key role in the healthcare continuum. MRI is a non-invasive, radiation-free imaging method that provides detailed images of the body, helping to identify and distinguish potential abnormalities in organs and tissues. This supports physicians in answering critical medical questions related to the detection and monitoring of diseases. About Radiology at Bayer Building on a century of expertise, Bayer is committed to innovative products and high-quality services in diagnostic imaging to enhance patient care. Its leading radiology portfolio features contrast agents and devices for precise administration across modalities including computed tomography (CT), X-ray and magnetic resonance imaging (MRI), and positron emission tomography (PET). Bayer's comprehensive offerings also include informatics solutions and a medical imaging platform with digital and artificial intelligence (AI) powered applications. In 2024, Bayer's radiology products generated €2.1 billion in sales. Bayer continues to advance research and innovation in medical imaging, including the integration of AI. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 'Health for all, Hunger for none,' the company's products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.
Business Insider
14-06-2025
- Business
- Business Insider
KalVista says FDA will not meet PDUFA goal date for Sebetralstat NDA
KalVista (KALV) Pharmaceuticals nnounced that the U.S. Food and Drug Administration has notified the company that it will not meet the PDUFA goal date for the New Drug Application for sebetralstat, the company's investigational oral on-demand treatment for hereditary angioedema. The FDA notified the company on June 13, 2025, that the previously disclosed June 17, 2025 PDUFA goal date will not be met due to heavy workload and limited resources. The FDA indicated that it expects to deliver a decision within approximately four weeks. The FDA has not requested additional data or studies and has not raised any concerns regarding the safety, efficacy or approvability of sebetralstat. KalVista has addressed all prior information requests in a timely manner, and the company believes the only remaining item under FDA review is the finalization of the labelling. 'We are disappointed by this delay, most importantly because we know how much people living with HAE are looking forward to an oral on-demand option to treat their HAE attacks,' said Ben Palleiko, CEO of KalVista. 'At the same time, we remain confident in the near-term approval of sebetralstat. We are continuing to work closely with the FDA to support the completion of their review. Our commitment to bringing this important therapy to people living with HAE remains unwavering.'
Cision Canada
13-06-2025
- Business
- Cision Canada
DRI Healthcare Trust Comments on FDA Delay for KalVista Pharmaceuticals' Sebetralstat PDUFA Goal Date Due to FDA Resourcing Constraints
- FDA decision anticipated within four weeks - TORONTO, June 13, 2025 /CNW/ - DRI Healthcare Trust (TSX: (TSX: DHT.U) (the "Trust") today announced KalVista Pharmaceuticals ("KalVista") has disclosed that it has received notice from the U.S. Food and Drug Administration ("FDA") that the FDA will not meet the June 17, 2025 PDUFA goal date for the New Drug Application (NDA) for sebetralstat, due to a heavy workload and agency resourcing issues. KalVista commented that the FDA has not requested additional data or studies and has communicated to KalVista that it anticipates delivering a decision within approximately four weeks. KalVista issued its own press release with the announcement earlier today (link). About DRI Healthcare Trust The Trust is managed by DRI Capital Inc., a pioneer in global pharmaceutical royalty monetization. Since its initial public offering in 2021, the Trust has deployed more than $1.0 billion, acquiring more than 25 royalties on 20-plus drugs, including Eylea, Orserdu, Omidria, Spinraza, Stelara, Vonjo, Zejula and Zytiga. The Trust's units are listed and trade on the Toronto Stock Exchange in Canadian dollars under the symbol " and in U.S. dollars under the symbol "DHT.U". To learn more, visit or follow us on LinkedIn. SOURCE DRI Healthcare Trust
