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Rare Disease Supporters Suggest Permanent PRV Program, Even If It May Concern FDA

 
• By Derrick Gingery

Non-profit biotech founder says permanent pediatric rare disease priority review voucher program will boost incentive, but FDA is concerned about resource drain vouchers cause.

Capitol House

Rare disease advocates want to add more certainty to the pediatric rare disease priority review voucher program, although that may put them at odds with the US FDA.

The pediatric rare disease priority review voucher program has generated hundreds of millions for sponsors, but the program will sunset...

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More from Rare Diseases

US FDA’s ‘Plausible Mechanism’ Pathway: A Platform-Based Approach Not Just For Rare Diseases

 
• By Sue Sutter

Manufacturers who succeed in treating several patients with bespoke therapies could leverage platform data to gain marketing approval for similar products in additional conditions, FDA leaders say in a NEJM article that describes five prerequisites for use of the new pathway.

Biohaven’s Troriluzole And The Era Of Real-Time Complete Response Letters

 
• By Michael McCaughan

US FDA’s rejection of Biohaven’s troriluzole is a huge disappointment for patients with SCA and the company. But it is also a notable example of how communication strategies are changing now that FDA is making its ‘complete response’ letters public.

Patient Perception-Of-Change Videos Not Efficacy Evidence For Stealth Barth Syndrome Drug

 
• By Sue Sutter

FDA reviewers cited several limitations of the videos, including a lengthy recall period, use of the term “today” in questions, and a lack of standardization in interview conduct.

Forzinity Chronology: Stealth’s Barth Syndrome Drug Bounced Around US FDA Review Divisions

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the clinical development and US FDA review timeline for Stealth's elamipretide for Barth syndrome.

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‘Why We Would Do It Again’ – Gilead & Roche On Pioneering Global Initiative For Manufacturing Change

Gilead and Roche reflect on their experiences with a global initiative that delivered aligned regulatory decisions through joint hybrid inspections and synchronized review of post-approval changes.

Novel Alzheimer’s Drug Blarcamesine Scheduled For High-Stakes EMA Oral Explanation

 
• By Neena Brizmohun

The EMA has scheduled oral explanation meetings this week for Anavex’s blarcamesine and three other products that are nearing the end of the regulatory review cycle. These meetings usually represent the final chance for sponsors to persuade the agency that their product merits approval.

Sponsored by

The British Pharmacopoeia: Setting standards that are trusted worldwide

In this exclusive article with Peter Crowley, Secretary and Scientific Director of the BP Commission, you’ll discover how the BP is preparing for its landmark 2026 edition while strengthening its influence as a global bridge between science, regulation, and industry.