Rare Pediatric Disease Voucher Program Gains Temporary Extension From House Committee
US incentive program would gain only four more years, despite calls for a permanent extension.
You may also be interested in...
US FDA received 110 designation requests in July, a high mark that reflects the 60 days needed to review such requests ahead of the 30 September statutory expiration; agency is allowing email submission of RPD and orphan drug designation requests during the COVID-19 pandemic and expects to roll out an online portal for orphan designation requests later this year.
Marinus Pharmaceuticals has transformed its finances and the prospects for its R&D pipeline over the past 12 months, and has just reported positive top-line results from the Phase III MARIGOLD study of its lead compound, ganaxolone.
Chair of the House Energy and Commerce Committee raises questions about the priority review voucher program’s value, suggesting another limited extension may be upcoming.