Travere Therapeutics and CSL Vifor Announce Standard EU Approval of FILSPARI

SAN DIEGO & ST. GALLEN, Switzerland--(BUSINESS WIRE)--Travere Therapeutics, Inc., (NASDAQ: TVTX) and CSL Vifor are pleased to announce that the European Commission has approved the conversion of the conditional marketing approval (CMA) into a standard marketing authorization (MA) for FILSPARI for the treatment of adults with primary IgA nephropathy with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). Standard MA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.
'The European Commission's standard approval of FILSPARI is a meaningful step forward for people living with IgA nephropathy across Europe,' said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. 'This decision not only validates the strength of the Phase 3 PROTECT Study results but also reinforces our deep commitment to this rare kidney disease community. We remain dedicated to working with our partners, regulators, and healthcare providers to expand access and improve outcomes for those affected by IgAN.'
'The decision by the European Commission is an important advancement for people living with IgAN in the EU,' said Vinicius Gomes De Lima, M.D., head of global medical affairs at CSL Vifor. 'The standard approval, granted without changes to the indication, underscores the value of our clinical data, the dedication of our teams, and our ongoing commitment to deliver on our promise for patients. We look forward to continuing working closely with healthcare professionals, patient communities, and regulatory bodies to ensure access to FILSPARI across Europe.'
The European Commission's decision follows the Committee for Medicinal Products for Human Use (CHMP) recommendation from February 2025 to convert the CMA to standard MA. The approval is based on a comprehensive clinical data set, including positive confirmatory results from the pivotal Phase 3 PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan.
As a result of the standard approval, the Company expects to receive a $17.5 million milestone payment from CSL Vifor, and the Company remains eligible to receive additional milestone payments related to market access and sales-based achievements for FILSPARI.
FILSPARI is the only Dual Endothelin Angiotensin Receptor Antagonist (DEARA), a non-immunosuppressive therapy for the treatment of IgAN approved in Europe and is currently available in Germany, Austria and Switzerland.
In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) converted the conditional FILSPARI approval to standard approval as of April 15, 2025. FILSPARI is indicated for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥ 0.75 g/g); the indication is unchanged in the UK.
About Travere Therapeutics
At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com.
About CSL Vifor
CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care).
The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, cslvifor.com.
About IgA Nephropathy (IgAN)
IgAN, also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure.
While rare, IgAN is the most common type of primary glomerular disease worldwide and a leading cause of kidney failure. IgAN is estimated to affect up to 250,000 people in the licensed territories (Europe, Australia and New Zealand).
About the PROTECT Study
The PROTECT Study is one of the largest interventional studies to date in IgA nephropathy (IgAN) and the only head-to-head vs. comparator trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of sparsentan, compared to 300 mg of irbesartan (an angiotensin II receptor blocker(ARB)), in 404 patients ages 18 years and up with IgA nephropathy and persistent proteinuria despite receiving at least 50% of maximum label dose and maximally tolerated angiotensin-converting enzyme (ACE) inhibitors or ARB therapy.
The PROTECT study met its primary endpoint at the pre-specified interim analysis with statistical significance. After 36 weeks of treatment, patients receiving FILSPARI achieved a mean reduction in proteinuria from baseline of 49.8%, compared to a mean reduction in proteinuria from baseline of 15.1% for irbesartan-treated patients. The two-year confirmatory results from the study showed treatment with FILSPARI achieved statistical significance on the eGFR chronic slope endpoint versus irbesartan and demonstrated clinically meaningful kidney function preservation. eGFR is a blood test that measure how well kidneys filter waste products from blood. Treatment emergent adverse events were well-balanced between sparsentan and irbesartan, except for dizziness and hypotension.
About FILSPARI (sparsentan)
FILSPARI is an innovative, non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist with high selectivity for the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R).
FILSPARI was developed by Travere Therapeutics and has been granted Orphan Drug Designation for the treatment of IgA nephropathy in the UK, Europe and the U.S. FILSPARI is currently available in the U.S. and first markets in Europe. CSL Vifor has been granted exclusive commercialization rights for FILSPARI in Europe, Australia and New Zealand.
For more information, please refer to the Summary of Product Characteristics (SmPC).
FILSPARI ® (sparsentan) U.S. Indication
FILSPARI (sparsentan) is indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: HEPATOTOXICITY AND EMBRYO-FETAL TOXICITY
Because of the risks of hepatotoxicity and birth defects, FILSPARI is available only through a restricted program called the FILSPARI REMS. Under the FILSPARI REMS, prescribers, patients and pharmacies must enroll in the program.
Hepatotoxicity
Some Endothelin Receptor Antagonists (ERAs) have caused elevations of aminotransferases, hepatotoxicity, and liver failure. In clinical studies, elevations in aminotransferases (ALT or AST) of at least 3-times the Upper Limit of Normal (ULN) have been observed in up to 3.5% of FILSPARI-treated patients, including cases confirmed with rechallenge.
Measure transaminases and bilirubin before initiating treatment and monthly for the first 12 months, and then every 3 months during treatment. Interrupt treatment and closely monitor patients who develop aminotransferase elevations more than 3x ULN.
FILSPARI should generally be avoided in patients with elevated aminotransferases (>3x ULN) at baseline because monitoring for hepatotoxicity may be more difficult and these patients may be at increased risk for serious hepatotoxicity.
Embryo-Fetal Toxicity
FILSPARI can cause major birth defects if used by pregnant patients based on animal data. Therefore, pregnancy testing is required before the initiation of treatment, during treatment and one month after discontinuation of treatment with FILSPARI. Patients who can become pregnant must use effective contraception before the initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI.
Contraindications
FILSPARI is contraindicated in patients who are pregnant. Do not coadminister FILSPARI with angiotensin receptor blockers (ARBs), ERAs, or aliskiren.
Warnings and Precautions
Hepatotoxicity: Elevations in ALT or AST of at least 3-fold ULN have been observed in up to 3.5% of FILSPARI-treated patients, including cases confirmed with rechallenge. While no concurrent elevations in bilirubin >2-times ULN or cases of liver failure were observed in FILSPARI-treated patients, some ERAs have caused elevations of aminotransferases, hepatotoxicity, and liver failure. To reduce the risk of potential serious hepatotoxicity, measure serum aminotransferase levels and total bilirubin prior to initiation of treatment and monthly for the first 12 months, then every 3 months during treatment.
Advise patients with symptoms suggesting hepatotoxicity (nausea, vomiting, right upper quadrant pain, fatigue, anorexia, jaundice, dark urine, fever, or itching) to immediately stop treatment with FILSPARI and seek medical attention. If aminotransferase levels are abnormal at any time during treatment, interrupt FILSPARI and monitor as recommended.
Consider re-initiation of FILSPARI only when hepatic enzyme levels and bilirubin return to pretreatment values and only in patients who have not experienced clinical symptoms of hepatotoxicity. Avoid initiation of FILSPARI in patients with elevated aminotransferases (>3x ULN) prior to drug initiation because monitoring hepatotoxicity in these patients may be more difficult and these patients may be at increased risk for serious hepatotoxicity.
Embryo-Fetal Toxicity: FILSPARI can cause fetal harm when administered to a pregnant patient and is contraindicated during pregnancy. Advise patients who can become pregnant of the potential risk to a fetus. Obtain a pregnancy test prior to initiation of treatment with FILSPARI, monthly during treatment, and one month after discontinuation of treatment. Advise patients who can become pregnant to use effective contraception prior to initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI.
FILSPARI REMS: Due to the risk of hepatotoxicity and embryo-fetal toxicity, FILSPARI is available only through a restricted program called the FILSPARI REMS. Prescribers, patients, and pharmacies must be enrolled in the REMS program and comply with all requirements (www.filsparirems.com).
Hypotension: Hypotension has been observed in patients treated with ARBs and ERAs. There was a greater incidence of hypotension-associated adverse events, some serious, including dizziness, in patients treated with FILSPARI compared to irbesartan. In patients at risk for hypotension, consider eliminating or adjusting other antihypertensive medications and maintaining appropriate volume status. If hypotension develops, despite elimination or reduction of other antihypertensive medications, consider a dose reduction or dose interruption of FILSPARI. A transient hypotensive response is not a contraindication to further dosing of FILSPARI, which can be given once blood pressure has stabilized.
Acute Kidney Injury: Monitor kidney function periodically. Drugs that inhibit the renin-angiotensin system (RAS) can cause kidney injury. Patients whose kidney function may depend in part on the activity of the RAS (e.g., patients with renal artery stenosis, chronic kidney disease, severe congestive heart failure, or volume depletion) may be at particular risk of developing acute kidney injury on FILSPARI. Consider withholding or discontinuing therapy in patients who develop a clinically significant decrease in kidney function while on FILSPARI.
Hyperkalemia: Monitor serum potassium periodically and treat appropriately. Patients with advanced kidney disease, taking concomitant potassium-increasing drugs (e.g., potassium supplements, potassium-sparing diuretics), or using potassium-containing salt substitutes are at increased risk for developing hyperkalemia. Dosage reduction or discontinuation of FILSPARI may be required.
Fluid Retention: Fluid retention may occur with ERAs, and has been observed in clinical studies with FILSPARI. FILSPARI has not been evaluated in patients with heart failure. If clinically significant fluid retention develops, evaluate the patient to determine the cause and the potential need to initiate or modify the dose of diuretic treatment then consider modifying the dose of FILSPARI.
Most common adverse reactions
The most common adverse reactions (≥5%) are hyperkalemia, hypotension (including orthostatic hypotension), peripheral edema, dizziness, anemia, and acute kidney injury.
Drug interactions
Renin-Angiotensin System (RAS) Inhibitors and ERAs: Do not coadminister FILSPARI with ARBs, ERAs, or aliskiren due to increased risks of hypotension, syncope, hyperkalemia, and changes in renal function (including acute renal failure).
Strong and Moderate CYP3A Inhibitors: Avoid concomitant use of FILSPARI with strong CYP3A inhibitors. If a strong CYP3A inhibitor cannot be avoided, interrupt FILSPARI treatment. When resuming treatment with FILSPARI, consider dose titration. Monitor blood pressure, serum potassium, edema, and kidney function regularly when used concomitantly with moderate CYP3A inhibitors. Concomitant use with a strong CYP3A inhibitor increases sparsentan exposure which may increase the risk of FILSPARI adverse reactions.
Strong CYP3A Inducers: Avoid concomitant use with a strong CYP3A inducer. Concomitant use with a strong CYP3A inducer decreases sparsentan exposure which may reduce FILSPARI efficacy.
Antacids and Acid Reducing Agents: Administer FILSPARI 2 hours before or after administration of antacids. Avoid concomitant use of acid reducing agents (histamine H2 receptor antagonist and PPI proton pump inhibitor) with FILSPARI. Sparsentan exhibits pH-dependent solubility. Antacids or acid reducing agents may decrease sparsentan exposure which may reduce FILSPARI efficacy.
Non-Steroidal Anti-Inflammatory Agents (NSAIDs), Including Selective Cyclooxygenase-2 (COX-2) Inhibitors: Monitor for signs of worsening renal function with concomitant use with NSAIDs (including selective COX-2 inhibitors). In patients with volume depletion (including those on diuretic therapy) or with impaired kidney function, concomitant use of NSAIDs (including selective COX-2 inhibitors) with drugs that antagonize the angiotensin II receptor may result in deterioration of kidney function, including possible kidney failure.
CYP2B6, 2C9, and 2C19 Substrates: Monitor for efficacy of concurrently administered CYP2B6, 2C9, and 2C19 substrates and consider dosage adjustment in accordance with the Prescribing Information. Sparsentan decreases exposure of these substrates, which may reduce efficacy related to these substrates.
P-gp and BCRP Substrates: Avoid concomitant use of sensitive substrates of P-gp and BCRP with FILSPARI. Sparsentan may increase exposure of these transporter substrates, which may increase the risk of adverse reactions related to these substrates.
Agents Increasing Serum Potassium: Monitor serum potassium frequently in patients treated with FILSPARI and other agents that increase serum potassium. Concomitant use of FILSPARI with potassium-sparing diuretics, potassium supplements, potassium-containing salt substitutes, or other drugs that raise serum potassium levels may result in hyperkalemia.
Please see the full Prescribing Information, including BOXED WARNING, for additional Important Safety Information.
Forward Looking Statements
This press release contains 'forward-looking statements' as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words 'on-track,' 'positioned,' 'look forward to,' 'will,' 'would,' 'may,' 'might,' 'believes,' 'anticipates,' 'plans,' 'expects,' 'intends,' 'potential,' or similar expressions. In addition, expressions of strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: statements and expectations regarding the expansion of access to FILSPARI in Europe and improvement in outcomes for those affected by IgAN; statements regarding future milestone payments; and statements related to the estimated size of patient populations. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the regulatory review and approval process, as well as risks and uncertainties associated with the continued commercial launch of FILSPARI in IgAN. The Company also faces risks and uncertainties related to its sNDA for FILSPARI in FSGS, including the timing and outcome thereof. There is no guarantee that the FDA will accept the sNDA for filing, grant priority review of the sNDA or grant approval of FILSPARI for FSGS on the anticipated timeline, or at all. The Company also faces risks and uncertainties related to its business and finances in general, the success of its commercial products, risks and uncertainties associated with its preclinical and clinical stage pipeline, risks and uncertainties associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. Specifically, the Company faces risks associated with the timing and potential outcome of its and its partners' clinical studies, market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, risks related to the challenges of manufacturing scale-up, risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI, and risks and uncertainties related to the new administration, including but not limited to risks and uncertainties related to tariffs and the funding, staffing and prioritization of resources at government agencies including the FDA. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading 'Risk Factors', as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.

Try Our AI Features

Explore what Daily8 AI can do for you:

Learn with AIFact CheckingText to SpeechAI Summary

Related Articles

Associated Press

3 hours ago

• Associated Press

Ono Pharmaceutical and Vertex Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea

OSAKA, Japan & BOSTON--(BUSINESS WIRE)--Jun 23, 2025-- Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; 'Ono') and Vertex Pharmaceuticals Incorporated (Headquarters: Boston, MA, U.S.; CEO: Reshma Kewalramani, M.D.; 'Vertex') today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other serious B cell-mediated diseases. Under the terms of the agreement, Ono will pay Vertex an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. 'Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,' said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. 'We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.' 'Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure, and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.' The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. View source version on CONTACT: Ono Pharmaceutical Co., Ltd. Corporate Communications [email protected] more information: Vertex Pharmaceuticals Incorporated Investors: [email protected]: [email protected] or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 KEYWORD: NORTH AMERICA UNITED STATES SOUTH KOREA ASIA PACIFIC JAPAN MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Ono Pharmaceutical Co., Ltd. Copyright Business Wire 2025. PUB: 06/23/2025 04:00 AM/DISC: 06/23/2025 04:02 AM

Associated Press

3 hours ago

• Associated Press

Vertex and Ono Pharmaceutical Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea

BOSTON & OSAKA, Japan--(BUSINESS WIRE)--Jun 23, 2025-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Ono Pharmaceutical Co., Ltd. (OTCMKTS: OPHLY) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other B cell-mediated diseases. Under the terms of the agreement, Vertex will receive an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. 'Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.' 'Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,' said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. 'We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.' About Povetacicept Povetacicept is a recombinant fusion protein therapeutic and a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation and/or survival of B cells, T cells and innate immune cells. Based upon an engineered TACI (transmembrane activator and calcium modulator ligand interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious B cell-mediated diseases including other autoimmune kidney diseases and autoimmune cytopenias. About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan. IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years. There are no approved therapies that specifically target the underlying cause of IgAN. About Primary Membranous Nephropathy (pMN) Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 150,000 people diagnosed in the U.S. and Europe. It is estimated that there are approximately 6,000 diagnosed patients with pMN in Japan. pMN is a rare glomerular disease that occurs when the body generates an abnormal immune response, including autoantibodies, against proteins that are part of the kidney. Autoantibodies trigger damage and inflammation, especially within the glomeruli (the parts of the kidney that filter blood), impairing the kidneys' ability to properly filter waste and fluid, eventually causing progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN. About RAINIER RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating urine protein to creatinine ratio (UPCR) for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104. The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. About RUBY-3 RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, primary membranous nephropathy, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. About Ono Pharmaceutical Co., Ltd Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of 'Dedicated to the Fight against Disease and Pain,' Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at Vertex Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani M.D., and Toichi Takino, in this press release, and statements about the terms of and expectations for Vertex's collaboration with Ono, statements about potential benefits and results that may be achieved through the collaboration, statements regarding the future activities of the parties pursuant to the collaboration, including Ono's help to advance clinical trials and Ono's responsibility to obtain marketing authorizations in Japan and South Korea and to commercialize povetacicept in the regions, statements regarding upfront and milestone payments, and potential royalties on future products, and statements about Vertex's plans and expectations for the RAINIER and RUBY-3 clinical trials and potential plans to seek accelerated approval in the U.S. based on interim analysis from the RAINIER trial. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of the collaboration between Vertex and Ono may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at and on the SEC's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Ono Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice. View source version on CONTACT: Vertex Pharmaceuticals Incorporated Investors: [email protected] Media: [email protected] or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 Ono Pharmaceutical Co., Ltd. Corporate Communications [email protected] KEYWORD: NORTH AMERICA UNITED STATES SOUTH KOREA ASIA PACIFIC JAPAN MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Vertex Pharmaceuticals Incorporated Copyright Business Wire 2025. PUB: 06/23/2025 04:00 AM/DISC: 06/23/2025 04:02 AM

Hamilton Spectator

5 hours ago

• Hamilton Spectator

Prime Minister Carney in Brussels today for EU-Canada summit

BRUSSELS - Prime Minister Mark Carney is in Belgium today, where he visited a military cemetery before meeting with European Union leaders at an EU-Canada summit. Carney said on social media Sunday that he was in Brussels to launch 'a new era of partnership' between Canada and the European Union for the benefit of workers, businesses and security 'on both sides of the Atlantic.' Carney started the day with a visit to the Antwerp Schoonselhof Military Cemetery where 348 Canadian soldiers are buried. Later, he is expected to meet with Belgian Prime Minister Bart De Wever, European Council President António Costa and European Commission President Ursula von der Leyen. Carney posted on social media early on Monday that he spoke with U.S. President Donald Trump overnight, noting the conversation addressed the need to de-escalate the conflict in the Middle East, their shared commitment of a stronger NATO and progress in ongoing trade talks between Canada and the United States. At the EU-Canada summit, Foreign Affairs Minister Anita Anand and Defence Minister David McGuinty are expected to sign a security and defence agreement with the European bloc in what one European official described last week as one of the most ambitious deals the continent's powers have ever signed with a third country. The security and defence agreement aims to open the door to Canada's participation in the joint purchase of weapons with European countries. It will also lead to Canada's participation in the ReArm Europe initiative, allowing Canada to access a 150-billion-euro program for defence procurement, called Security Action for Europe. Canada will need to sign a second agreement with the European Commission before it can take part in the program. A government official briefing reporters on the trip said the partnership is expected to make procurement easier and more affordable, while also allowing Canada to diversify the sources of equipment. At the EU-Canada summit, leaders are also expected to issue a joint statement to underscore a willingness for continued pressure on Russia to end its war on Ukraine, including through further sanctions, and call for an immediate and permanent ceasefire in Gaza. The joint statement is also expected to touch on climate change, trade and digital and tech policy. Leaders at the EU-Canada summit are also slated to discuss global trade and commit to working towards full ratification and implementation of the Comprehensive Economic and Trade Agreement, the Canada-Europe free trade deal known as CETA. The pact took effect provisionally in 2017, and most of its contents now apply. But all EU countries need to approve CETA before it can take full effect, with 10 members still left to ratify the deal. Carney, Costa and von der Leyen are scheduled to hold a joint press conference in the evening. On Tuesday, Carney travels to The Hague for the NATO summit. The international meetings come as Canada looks to reduce its defence procurement reliance on the United States due to strained relations over tariffs and U.S. President Donald Trump's repeated talk about Canada becoming a U.S. state. This report by The Canadian Press was first published June 23, 2025.