Medicus Pharma Submits Phase 2b Protocol to FDA for SkinJect in Gorlin Syndrome NDA Push

Medicus Pharma's submission of Protocol SKNJCT-005 to the FDA signals a direct move toward NDA-enabling data for SkinJect® in Gorlin Syndrome, an orphan indication where no lesion-directed therapy currently holds agency approval. For regulatory affairs leads tracking rare disease pathways, the open-label Phase 2b design reflects a calculated alignment with FDA's accelerated frameworks for unmet-need populations.

Gorlin Syndrome, or Nevoid Basal Cell Carcinoma Syndrome (NBCCS), is a rare autosomal dominant disorder driven by mutations in the Hedgehog signaling pathway. Affected patients can develop dozens to more than one thousand basal cell carcinomas across a lifetime, with lesions frequently appearing in childhood and adolescence. The U.S. patient population is estimated at 6,000 to 12,000 individuals, meeting the threshold for Orphan Drug Designation under 21 CFR Part 316.

The submitted protocol sits under Medicus's existing Investigational New Drug (IND) application. The Phase 2b, open-label study is structured to generate efficacy and safety data intended to support a future NDA submission. Medicus applied for Orphan Drug Designation in April 2026 and has indicated it intends to file for a Rare Pediatric Disease Priority Review Voucher before the close of Q2 2026, a designation that would carry meaningful review timeline and commercial implications if granted.

SkinJect® is a microneedle-based chemotherapy platform designed for localized, lesion-directed delivery. The platform's ability to treat multiple lesions simultaneously is central to the clinical rationale in Gorlin Syndrome, where the surgical burden across a patient's lifetime is substantial. No FDA-approved lesion-directed therapy currently exists for this population, a gap that frames the regulatory opportunity Medicus is pursuing.

For QA and manufacturing leads, the transition into registrational development under an existing IND means process validation and GMP readiness timelines will need to align with the Phase 2b enrollment schedule. ICH Q10-compliant pharmaceutical quality systems will be under scrutiny as the program moves toward data packages intended to support NDA filing.

The outcome of the Orphan Drug Designation application and the Rare Pediatric Disease Priority Review Voucher filing, both expected to resolve within the current regulatory calendar, will set the near-term pace for the SkinJect® Gorlin Syndrome program.

Source: Medicus Pharma Ltd. via GlobeNewswire, June 3, 2026.