Medicus Pharma Ltd. (NASDAQ: MDCX) submitted an orphan drug designation application to the U.S. Food and Drug Administration for its SkinJect treatment in patients with Gorlin syndrome, according to a company statement.

SkinJect is a doxorubicin-containing dissolvable microneedle array designed for direct intradermal delivery into basal cell carcinoma lesions. The treatment targets Gorlin syndrome, a rare genetic disorder that causes patients to develop multiple, recurrent skin cancers throughout their lifetime.

Gorlin syndrome affects an estimated 11,000 patients in the United States, well below the 200,000 threshold required for orphan designation. Patients with the condition may develop dozens to over 1,000 basal cell carcinomas over their lifetime, often beginning in childhood.

The FDA has not approved any therapies specifically for basal cell carcinoma in Gorlin syndrome patients. Current treatments rely primarily on surgical excision or Mohs surgery, while systemic therapies like Hedgehog inhibitors face tolerability issues and tumor recurrence upon discontinuation.

SkinJect has been evaluated in Phase 1 and Phase 2 clinical studies in basal cell carcinoma patients. The studies showed a favorable safety profile with adverse events primarily limited to treatment sites, no serious adverse events or dose limiting toxicities, and evidence of clinical clearance and histological complete responses in treated lesions.

If granted, orphan drug designation would provide seven years of U.S. market exclusivity upon approval, potential eligibility for tax credits and fee waivers, and enhanced regulatory interaction with the FDA.

The microneedle array penetrates the skin and dissolves in place, releasing doxorubicin locally for direct cytotoxic tumor cell killing and induction of immunogenic cell death and local immune activation.