- Commercial-stage biotech BioCryst Pharmaceuticals, Inc. ( NASDAQ: BCRX ) announced Wednesday that the FDA granted the orphan drug designation for its experimental drug BCX9250 for the treatment of the rare genetic disorder fibrodysplasia ossificans progressiva (FOP).
- With its orphan drug designation, the FDA aims to offer financial incentives to drug developers targeting rare diseases and conditions.
- In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers can claim seven years of marketing exclusivity upon regulatory approval of orphan drugs.
- BCRX has already shared Phase 1 data for BCX9250, noting that the candidate was safe and well tolerated at all doses with the potential for once-daily dosing.
- An ultra-rare hereditary disorder, FOP is characterized by irregular bone formation outside the normal skeleton leading to deformities, restrictions on movement, and eventually death.
- In June, BCRX announced that the FDA awarded Fast Track designation for BCX9250 for the same indication.
For further details see:
BioCryst wins FDA’s Orphan Drug status for bone disorder therapy