Medicus Pharma Ltd. (NASDAQ: MDCX) submitted a Rare Pediatric Disease Designation request to the U.S. Food and Drug Administration for SkinJect, its investigational doxorubicin-containing microneedle array patch, to treat basal cell carcinoma in patients with Gorlin Syndrome.

The submission follows the company's previously announced Orphan Drug Designation application and registrational study design, which remains under FDA review. Medicus developed its Gorlin Syndrome program through engagement with clinical experts and the Gorlin Syndrome Alliance.

Gorlin Syndrome is a rare genetic disorder that affects approximately 1 in 30,000 to 60,000 individuals worldwide. The condition causes patients to develop dozens to thousands of basal cell carcinomas during their lifetime, often beginning in childhood and continuing throughout life. The estimated U.S. patient population ranges from 6,000 to 12,000 individuals.

SkinJect is designed to deliver doxorubicin directly into basal cell carcinoma lesions through localized intradermal administration. In a recently completed Phase 2 study, the 200-microgram treatment demonstrated clinical clearance rates of 64% and complete response rates of 55% among evaluable patients with nodular basal cell carcinoma.

If granted, the designation would support Medicus' regulatory strategy and could provide eligibility for a Rare Pediatric Disease Priority Review Voucher upon future FDA approval. The voucher could reduce FDA review time for a New Drug Application from approximately ten months to six months.

Additionally, if Orphan Drug Designation is granted and a marketing application is approved, SkinJect may become eligible for seven years of U.S. market exclusivity, waiver of certain FDA application fees, and additional regulatory incentives for rare disease therapies.

The information is based on a company press release statement.