Notizia - Recent Pharma, Healthcare and Biotech Happenings
Aug 26, 2025
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Madrigal Pharmaceuticals announced that the European Commission (EC) has granted conditional marketing authorization for REZDIFFRA, the first and only approved therapy in the European Union (EU) for adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis. The approval follows a positive CHMP opinion in June 2025 and is valid across all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway.
“This approval of REZDIFFRA marks a historic breakthrough for patients in Europe living with MASH, a serious and progressive liver disease,” said Bill Sibold, CEO of Madrigal. “The European labelling will set an important precedent, with no biopsy required to qualify for treatment and a clear focus on patients with moderate to advanced fibrosis (F2-F3), who are at significantly higher risk of liver-related mortality.”
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The EC’s decision was based on results from the pivotal phase III MAESTRO-NASH trial, where REZDIFFRA met both primary endpoints of fibrosis reduction and MASH resolution. The therapy also improved liver stiffness, fat, enzymes, and lipids, while enhancing quality of life. At one year, 91% of patients on REZDIFFRA 100 mg achieved improvement or stabilization of liver stiffness.
Frank Tacke, MD, PhD, Charité – Universitätsmedizin Berlin, stated: “The approval of REZDIFFRA is a transformational moment for the European MASH community. It is already included in European treatment guidelines, and with hundreds of patients enrolled in phase III studies, there is strong clinical experience supporting its use.” Madrigal plans to launch REZDIFFRA in Germany in Q4 2025, with broader rollout subject to national reimbursement decisions.
Valneva SE announced that the FDA has suspended the U.S. license for IXCHIQ, its chikungunya vaccine, after four new serious adverse events (SAEs) resembling chikungunya-like illness were reported. The suspension is effective immediately, requiring the company to halt shipments and sales in the U.S.
“This decision comes as the global threat of chikungunya continues to escalate,” said Thomas Lingelbach, CEO of Valneva. “We remain fully committed to maintaining access to IXCHIQ as a global health tool, particularly in endemic regions, and will continue working with partners to ensure the vaccine reaches those most in need.”
The move follows an earlier FDA action in August 2025 to lift a recommended pause on IXCHIQ use in people over 60, but updated VAERS data, including four additional SAEs outside the U.S., prompted a full license suspension. Three of the cases involved elderly patients aged 70–82, and one in a 55-year-old. Valneva stated these events are consistent with safety observations from clinical trials and post-marketing experience, particularly in older individuals with underlying conditions.
Valneva is assessing the potential financial impact of a permanent withdrawal but has not changed its revenue guidance. IXCHIQ generated €7.5 million of the company’s €91 million product sales in H1 2025, largely from emergency vaccine deliveries to La Réunion during a chikungunya outbreak.
Coya Therapeutics announced that the FDA has accepted its Investigational New Drug (IND) application for COYA 302, a proprietary immunomodulatory biologic combination therapy for amyotrophic lateral sclerosis (ALS). The clearance enables the initiation of a multicenter, double-blind, placebo-controlled phase II trial to evaluate the therapy’s safety and efficacy.
“The FDA’s acceptance of this IND marks a pivotal moment in Coya’s journey,” said Arun Swaminathan, CEO of Coya. “We are now preparing to initiate a well-powered phase II study and are grateful to the ALS community for their continued support of this important program.”
Milan Kalawadia, CEO North America at Dr. Reddy’s Laboratories (DRL), added: “This milestone further reinforces the strong scientific and strategic rationale behind our partnership with Coya. We are energized by the potential of COYA 302 as we move closer to offering a treatment for patients living with ALS.”
The IND approval also triggers a $4.2 million milestone payment from DRL to Coya, supporting advancement of the program.
Protagonist Therapeutics announced that the FDA has granted Breakthrough Therapy Designation to rusfertide, a potential first-in-class hepcidin-mimetic peptide, for the treatment of erythrocytosis in patients with polycythemia vera (PV). The designation builds on the therapy’s Orphan Drug and Fast Track statuses, offering multiple regulatory and development benefits.
“We are very pleased with the FDA’s decision, which underscores the potential of RUSFERTIDE to deliver substantial improvement over current PV therapies,” said Dinesh V. Patel, Ph.D., President and CEO of Protagonist. “We remain on track to submit an NDA for RUSFERTIDE by the end of this year.”
The FDA decision was supported by positive phase III VERIFY trial data, presented during the plenary session at the 2025 ASCO Annual Meeting. The 32-week results highlighted RUSFERTIDE’s ability to achieve hematocrit control, reduce phlebotomy dependence, and improve patient-reported outcomes including fatigue.
“The comprehensive data provide compelling evidence of RUSFERTIDE’s ability to address unmet needs in PV,” added Arturo Molina, M.D., M.S., CMO of Protagonist. The therapy is being co-developed with Takeda Pharmaceuticals under a global collaboration agreement, with Protagonist leading development through NDA filing.
Crinetics Pharmaceuticals announced that the FDA has granted Orphan Drug Designation (ODD) to Atumelnant, the first and only small-molecule ACTH receptor antagonist in clinical development, for the treatment of classic congenital adrenal hyperplasia (CAH). The designation provides key regulatory and financial incentives, including seven years of market exclusivity upon approval.
“This designation underscores the significant unmet need faced by patients with CAH,” said Dana Pizzuti, M.D., Chief Medical and Development Officer at Crinetics. “Atumelnant’s novel mechanism offers the potential to restore adrenal androgen balance, reduce glucocorticoid supplementation, and meaningfully improve patients’ quality of life.”
The decision builds on strong results from the phase II TouCAHn trial, which showed rapid and sustained biomarker improvements, including up to an 80% reduction in androstenedione, along with clinical benefits such as resumption of menses and reduced adrenal size. Crinetics expects to begin enrolling participants in the CALM-CAH phase III trial in adults and the BALANCE-CAH phase II/III trial in pediatrics in the second half of 2025.
Classic CAH, caused by impaired cortisol synthesis, remains an area of high unmet need. Current treatments rely on chronic glucocorticoid supplementation, often at supraphysiologic levels, leading to complications such as diabetes, cardiovascular disease, and osteoporosis. Crinetics aims for ATUMELNANT to provide a safer, more targeted approach to long-term disease management.
So, this was the pharma news — Madrigal’s REZDIFFRA won EU conditional approval for MASH with fibrosis; Valneva’s IXCHIQ had its U.S. license suspended after new SAE reports; Coya’s COYA 302 IND was accepted in ALS, enabling a phase II trial and a $4.2M milestone; Protagonist’s RUSFERTIDE earned FDA Breakthrough Therapy in PV on phase III VERIFY data; and Crinetics’ ATUMELNANT received FDA Orphan Drug status in CAH with pivotal starts slated for H2 2025. Net-net: major regulatory wins, one safety setback, and multiple catalysts advancing toward late-stage development.
Article in PDF
Aug 26, 2025
Table of Contents
Madrigal Pharmaceuticals announced that the European Commission (EC) has granted conditional marketing authorization for REZDIFFRA, the first and only approved therapy in the European Union (EU) for adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis. The approval follows a positive CHMP opinion in June 2025 and is valid across all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway.
“This approval of REZDIFFRA marks a historic breakthrough for patients in Europe living with MASH, a serious and progressive liver disease,” said Bill Sibold, CEO of Madrigal. “The European labelling will set an important precedent, with no biopsy required to qualify for treatment and a clear focus on patients with moderate to advanced fibrosis (F2-F3), who are at significantly higher risk of liver-related mortality.”
The EC’s decision was based on results from the pivotal phase III MAESTRO-NASH trial, where REZDIFFRA met both primary endpoints of fibrosis reduction and MASH resolution. The therapy also improved liver stiffness, fat, enzymes, and lipids, while enhancing quality of life. At one year, 91% of patients on REZDIFFRA 100 mg achieved improvement or stabilization of liver stiffness.
Frank Tacke, MD, PhD, Charité – Universitätsmedizin Berlin, stated: “The approval of REZDIFFRA is a transformational moment for the European MASH community. It is already included in European treatment guidelines, and with hundreds of patients enrolled in phase III studies, there is strong clinical experience supporting its use.” Madrigal plans to launch REZDIFFRA in Germany in Q4 2025, with broader rollout subject to national reimbursement decisions.
Valneva SE announced that the FDA has suspended the U.S. license for IXCHIQ, its chikungunya vaccine, after four new serious adverse events (SAEs) resembling chikungunya-like illness were reported. The suspension is effective immediately, requiring the company to halt shipments and sales in the U.S.
“This decision comes as the global threat of chikungunya continues to escalate,” said Thomas Lingelbach, CEO of Valneva. “We remain fully committed to maintaining access to IXCHIQ as a global health tool, particularly in endemic regions, and will continue working with partners to ensure the vaccine reaches those most in need.”
The move follows an earlier FDA action in August 2025 to lift a recommended pause on IXCHIQ use in people over 60, but updated VAERS data, including four additional SAEs outside the U.S., prompted a full license suspension. Three of the cases involved elderly patients aged 70–82, and one in a 55-year-old. Valneva stated these events are consistent with safety observations from clinical trials and post-marketing experience, particularly in older individuals with underlying conditions.
Valneva is assessing the potential financial impact of a permanent withdrawal but has not changed its revenue guidance. IXCHIQ generated €7.5 million of the company’s €91 million product sales in H1 2025, largely from emergency vaccine deliveries to La Réunion during a chikungunya outbreak.
Coya Therapeutics announced that the FDA has accepted its Investigational New Drug (IND) application for COYA 302, a proprietary immunomodulatory biologic combination therapy for amyotrophic lateral sclerosis (ALS). The clearance enables the initiation of a multicenter, double-blind, placebo-controlled phase II trial to evaluate the therapy’s safety and efficacy.
“The FDA’s acceptance of this IND marks a pivotal moment in Coya’s journey,” said Arun Swaminathan, CEO of Coya. “We are now preparing to initiate a well-powered phase II study and are grateful to the ALS community for their continued support of this important program.”
Milan Kalawadia, CEO North America at Dr. Reddy’s Laboratories (DRL), added: “This milestone further reinforces the strong scientific and strategic rationale behind our partnership with Coya. We are energized by the potential of COYA 302 as we move closer to offering a treatment for patients living with ALS.”
The IND approval also triggers a $4.2 million milestone payment from DRL to Coya, supporting advancement of the program.
Protagonist Therapeutics announced that the FDA has granted Breakthrough Therapy Designation to rusfertide, a potential first-in-class hepcidin-mimetic peptide, for the treatment of erythrocytosis in patients with polycythemia vera (PV). The designation builds on the therapy’s Orphan Drug and Fast Track statuses, offering multiple regulatory and development benefits.
“We are very pleased with the FDA’s decision, which underscores the potential of RUSFERTIDE to deliver substantial improvement over current PV therapies,” said Dinesh V. Patel, Ph.D., President and CEO of Protagonist. “We remain on track to submit an NDA for RUSFERTIDE by the end of this year.”
The FDA decision was supported by positive phase III VERIFY trial data, presented during the plenary session at the 2025 ASCO Annual Meeting. The 32-week results highlighted RUSFERTIDE’s ability to achieve hematocrit control, reduce phlebotomy dependence, and improve patient-reported outcomes including fatigue.
“The comprehensive data provide compelling evidence of RUSFERTIDE’s ability to address unmet needs in PV,” added Arturo Molina, M.D., M.S., CMO of Protagonist. The therapy is being co-developed with Takeda Pharmaceuticals under a global collaboration agreement, with Protagonist leading development through NDA filing.
Crinetics Pharmaceuticals announced that the FDA has granted Orphan Drug Designation (ODD) to Atumelnant, the first and only small-molecule ACTH receptor antagonist in clinical development, for the treatment of classic congenital adrenal hyperplasia (CAH). The designation provides key regulatory and financial incentives, including seven years of market exclusivity upon approval.
“This designation underscores the significant unmet need faced by patients with CAH,” said Dana Pizzuti, M.D., Chief Medical and Development Officer at Crinetics. “Atumelnant’s novel mechanism offers the potential to restore adrenal androgen balance, reduce glucocorticoid supplementation, and meaningfully improve patients’ quality of life.”
The decision builds on strong results from the phase II TouCAHn trial, which showed rapid and sustained biomarker improvements, including up to an 80% reduction in androstenedione, along with clinical benefits such as resumption of menses and reduced adrenal size. Crinetics expects to begin enrolling participants in the CALM-CAH phase III trial in adults and the BALANCE-CAH phase II/III trial in pediatrics in the second half of 2025.
Classic CAH, caused by impaired cortisol synthesis, remains an area of high unmet need. Current treatments rely on chronic glucocorticoid supplementation, often at supraphysiologic levels, leading to complications such as diabetes, cardiovascular disease, and osteoporosis. Crinetics aims for ATUMELNANT to provide a safer, more targeted approach to long-term disease management.
So, this was the pharma news — Madrigal’s REZDIFFRA won EU conditional approval for MASH with fibrosis; Valneva’s IXCHIQ had its U.S. license suspended after new SAE reports; Coya’s COYA 302 IND was accepted in ALS, enabling a phase II trial and a $4.2M milestone; Protagonist’s RUSFERTIDE earned FDA Breakthrough Therapy in PV on phase III VERIFY data; and Crinetics’ ATUMELNANT received FDA Orphan Drug status in CAH with pivotal starts slated for H2 2025. Net-net: major regulatory wins, one safety setback, and multiple catalysts advancing toward late-stage development.
