Sponsors May Race For Rare Pediatric Voucher Designations As Deadline Nears
US FDA program's new 2020 sunset could create a bolus of designation requests to ensure they can still get a voucher if it ends.
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Unless reauthorized by Congress, the rare pediatric disease program will begin to expire in September; renewal could bring bigger changes across the three PRV programs, including an FDA-requested clarification of biologic products eligible for vouchers and reforms suggested by sponsors and other stakeholders in a recent GAO report.
What Is A Rare Pediatric Disease? US FDA's Orphan Products Office Hopes To Update Guidance On Definition
The Office of Pediatric Therapeutics is working with the Office of Orphan Products Development to make rare pediatric disease determinations, which can help sponsors obtain a priority review voucher.
Capricor reported positive interim Phase I/II results for its cell therapy designed to treat cardiomyopathy in DMD patients and said it plans to seek regenerative medicine advanced technology, breakthrough or both designations from the US FDA.