Medicus Pharma Announces U.S. Food and Drug Administration (FDA) Submission of Rare Pediatric Disease Designation Request for SkinJect® in Gorlin Syndrome

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• Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age

  60% confidence

• If granted, the Rare Pediatric Disease Priority Review Voucher may reduce FDA review time for a future NDA from approximately ten months under standard review to approximately six months under priority review

  60% confidence

• SkinJect® Phase 2 study showed 64% clinical clearance and 55% complete response, supporting its potential as a non-surgical treatment for Gorlin Syndrome

  60% confidence

• Gorlin Syndrome represents a compelling development opportunity for SkinJect® given significant unmet medical need, recurring disease nature, lack of approved therapies for pediatric patients, and potential for lesion-directed non-surgical treatment

  60% confidence

• The 200µg D-MNA treatment arm demonstrated the strongest rates of clinical and histological clearance of 64% and 55% respectively among evaluable nodular BCC patients while maintaining a favorable safety and tolerability profile

  60% confidence

• SkinJect® and BCC/Gorlin Syndrome together represent a ~$2 billion annual market opportunity

  60% confidence

• Gorlin Syndrome affects approximately 1 in 30,000 to 60,000 individuals worldwide, corresponding to a U.S. patient population of approximately 6,000 to 12,000 individuals

  60% confidence

• SkinJect® may be uniquely positioned to address both pediatric and adult manifestations of Gorlin Syndrome through a repeatable, lesion-directed treatment approach

  60% confidence

• Teverelix® represents a ~$6 billion annual market opportunity

  60% confidence

• If Orphan Drug Designation is granted and a marketing application approved, SkinJect® may be eligible for seven years of U.S. market exclusivity, waiver of certain FDA application fees, and additional regulatory incentives

  60% confidence

Data points we hold from this source