FDA’s New “Plausible Mechanism Pathway” and Other Initiatives …
Nov 18, 2025 · The Plausible Mechanism Pathway, and other recent initiatives and guidance, reflect FDA’s awareness and renewed attention to addressing and mitigating some of the …
Drugmakers Wrestle With FDA Oversight of Rare Disease …
Drug companies that specialize in care for rare diseases are growing frustrated with the FDA, despite agency leadership’s proclaimed agenda to accelerate approvals and heighten flexibility …
Recent signals regarding rare disease drug development from …
May 8, 2025 · There continues to be an evolution in how the federal government and officials are looking at the challenge of evaluating and regulating the approval of drugs for ultra-rare diseases.
FDA Conditional Pathway Not Necessary, Rare Disease Experts
Jun 24, 2025 · At a BioSpace -moderated panel at BIO2025 last week, experts in drug development for rare and ultrarare diseases argued that an entirely new regulatory pathway …
Sarepta’s 40% Plunge and Accelerated Approval Crisis: What UCB’s Rare …
Nov 5, 2025 · All three Sarepta DMD drugs received accelerated approval based on increases in dystrophin protein production—a surrogate endpoint. The FDA granted approval contingent on …
FDA Accelerated Approval What’s Changing and Why It Matters for Rare ...
Sep 10, 2025 · These updates reflect the FDA’s efforts to enhance transparency in regulatory decision-making and recognize that recent initiatives, including updated guidance and public …
FDA’s Recent Guidance on Accelerated Approval and Implications for Rare ...
FDA’s recent guidance has significant implications for rare diseases. Patients with rare diseases often have limited treatment options, and the accelerated approval pathway is an...
Worth the risk: FDA must move more quickly on treatments for rare diseases
Aug 26, 2025 · FDA leadership has since partially reinstated a Duchenne gene therapy and offered accelerated reconsideration of Stealth’s Barth syndrome treatment. These are positive …
FDA signs off on Omeros' stem cell transplant drug Yartemlea
Dec 26, 2025 · The FDA has approved Omeros' Yartemlea as the first treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy.
Understanding FDA’s Accelerated Approval Reforms in Rare Diseases
This article unpacks the FDA’s draft guidance on Accelerated Approval, analyzing its impact on clinical evidence, trial design, and pharma accountability.