Orphan Drug & Rare Pediatric Disease Designations (incentives)
FDA Office of Orphan Products Development
Regulatory incentives rather than grants. Orphan Drug Designation brings 7-year marketing exclusivity, a 25% tax credit on qualified clinical trial costs, a PDUFA fee waiver, and eligibility for the FDA orphan grants. The Rare Pediatric Disease Priority Review Voucher program was reauthorized in Feb 2026 through September 2029: a sponsor winning approval receives a transferable voucher, and recent vouchers have sold for more than $100M.
BTHS meets the prevalence threshold for orphan designation, and a BTHS therapy approval would qualify for a rare pediatric disease voucher — which is a large part of why commercial sponsors engage with the disease at all.
Drug/biologic sponsors — companies or academic sponsors of INDs.
Designation requests go to OOPD on a rolling basis (no fee, no deadline) via FDA's designation request forms.
/grants/orphan-drug-and-rare-pediatric-disease-designations-incentives/
Last updated August 28, 2026 · Suggest an update to this entry