Rare Pediatric Disease Voucher Program Gains Temporary Extension From House Committee
Executive Summary
US incentive program would gain only four more years, despite calls for a permanent extension.
You may also be interested in...
Rare Pediatric Disease Designation Requests Skyrocket Ahead Of Voucher Program’s Sunset
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.
Rare Genetic Epilepsy Responsive To Marinus's Ganaxolone
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.
Rare Pediatric Priority Review Voucher Program Seems Headed For Short-Term Renewal
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.
Need a specific report? 1000+ reports available
Buy Reports
Register for our free email digests: