USFDAJun 15, 2026, 07:31 AM
Medicus Pharma Submits Rare Pediatric Disease Designation for SkinJect®
AI Summary
Medicus Pharma Ltd. announced the submission of a Rare Pediatric Disease Designation (RPDD) request to the U.S. FDA for SkinJect®, its investigational doxorubicin-containing microneedle array patch, for treating basal cell carcinoma in patients with Gorlin Syndrome. This follows a previous Orphan Drug Designation application and positive Phase 2 study results showing 64% clinical clearance and 55% complete response. If granted, RPDD could lead to a Priority Review Voucher, potentially accelerating FDA review, and Orphan Drug Designation could provide seven years of market exclusivity, addressing a significant unmet medical need for this rare disease.
Key Highlights
- Medicus Pharma submitted a Rare Pediatric Disease Designation (RPDD) request to the FDA for SkinJect®.
- SkinJect® is for the treatment of basal cell carcinoma in patients with Gorlin Syndrome.
- The RPDD submission follows a previously announced Orphan Drug Designation (ODD) application.
- Phase 2 study showed 64% clinical clearance and 55% complete response for SkinJect®.
- Gorlin Syndrome affects an estimated 6,000 to 12,000 individuals in the U.S.
- If granted, RPDD may provide eligibility for a Rare Pediatric Disease Priority Review Voucher.
- A Priority Review Voucher could reduce FDA review time from 10 months to 6 months.
- If ODD is granted, SkinJect® may receive 7 years of U.S. market exclusivity.
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