FDA Grants Rare Pediatric Disease Designation for CCP-020 for Treatment of Epidermolysis Bullosa
Summary:** Castle Creek Biosciences received FDA Rare Pediatric Disease Designation for CCP-020, a topical formulation of clobetasol propionate for epidermolysis bullosa (EB), a genetic disorder causing fragile skin and chronic blisters/ulcers. Affecting ~1 in 20,000 births, EB leads to painful wounds requiring daily bandaging; CCP-020 aims to reduce blistering and inflammation. The designation accelerates review and grants a priority review voucher upon approval, supporting development for this orphan condition with no curative treatments beyond supportive wound care.
Key Highlights:
- CCP-020: Topical clobetasol for EB blister reduction and wound prevention.
- Rare Designation: For conditions <200,000 U.S. cases; prioritizes review and incentives.
- EB Burden: Chronic wounds, infections, 30% mortality by age 35 from skin cancer.
- Development: Phase 2 trials ongoing; voucher aids funding for rare disease therapies.
- Implications: Advances EB wound management beyond dressings and pain control.
Keywords: CCP-020, epidermolysis bullosa, rare pediatric disease, clobetasol, EB blisters