Medicus Pharma Takes SkinJect Orphan Push to Capitol Hill

Medicus Pharma's direct engagement with senior House Energy and Commerce Committee members signals a calculated effort to use rare disease policy levers -- Orphan Drug Designation, registrational IND approval, and pediatric priority review vouchers -- to compress the regulatory timeline for SkinJect in Gorlin Syndrome. For regulatory affairs leads tracking orphan indication strategy, the approach illustrates how coordinated congressional outreach can run parallel to FDA pathway planning.

CEO Dr. Raza Bokhari and the Medicus leadership team met on Capitol Hill with Committee Chair Brett Guthrie, Health Subcommittee Vice Chair Diana Harshbarger, Energy and Commerce member John Joyce (Co-Chair, Congressional Skin Cancer Caucus), and Gus Bilirakis (Co-Chair, Congressional Rare Disease Caucus). Meetings were conducted in collaboration with the Gorlin Syndrome Alliance and focused on three regulatory objectives: Orphan Drug Designation for SkinJect, registrational IND approval for Gorlin Syndrome patients, and access to the rare disease pediatric FDA voucher program.

The unmet need framing is clinically specific. Gorlin Syndrome -- nevoid basal cell carcinoma syndrome -- is driven by mutations in the PTCH1 gene, causing dysregulation of the Hedgehog signaling pathway and early, recurrent tumor formation. Patients may develop dozens to over 1,000 basal cell carcinomas across a lifetime, often beginning in childhood. No FDA-approved therapies exist specifically for BCC in Gorlin Syndrome. Systemic Hedgehog inhibitors carry tolerability limitations and tumors recur upon discontinuation, leaving surgical excision and Mohs surgery as the primary standards of care. SkinJect is positioned as a localized, repeatable, non-surgical alternative.

Medicus states it is concurrently advancing regulatory and clinical planning toward a registrational development pathway, with stated intent to generate decision-grade data for expedited development. QA and regulatory teams at comparable rare disease sponsors will recognize the dual-track model: legislative relationship-building alongside IND-stage planning is increasingly standard practice for companies pursuing accelerated FDA pathways in high-burden orphan populations.

Source: Medicus Pharma press release via GlobeNewswire, April 27, 2026.