Medicus Pharma CEO meets with House lawmakers on rare disease treatment

Medicus Pharma Ltd. (NASDAQ: MDCX) announced that CEO Dr. Raza Bokhari and the company's leadership team met with lawmakers on Capitol Hill to discuss support for accelerated access to its SkinJect treatment for Gorlin Syndrome patients.

The meetings included discussions with Representative Brett Guthrie, Chair of the House Energy & Commerce Committee, Representative John Joyce, Co-Chair of the Congressional Skin Cancer Caucus, Representative Diana Harshbarger, Vice Chair of the House Energy & Commerce Subcommittee on Health, and Representative Gus Bilirakis, Co-Chair of the Congressional Rare Disease Caucus.

The company sought support for three initiatives: Orphan Drug Designation for SkinJect for Gorlin Syndrome, registrational Investigation New Drug approval for Gorlin Syndrome patients, and inclusion in the rare disease pediatric FDA voucher program.

Gorlin Syndrome is a rare inherited condition caused by mutations in the PTCH1 gene. Patients may develop dozens to over 1,000 basal cell carcinomas over their lifetime, often beginning in childhood. The condition requires repeated surgical interventions.

According to the company, no FDA-approved therapies exist specifically for basal cell carcinoma in Gorlin Syndrome. Current treatments rely on surgical excision or Mohs surgery, while systemic therapies face limitations regarding tolerability and recurrence upon discontinuation.

"Our engagement with policymakers reflects a clear alignment around accelerating access to innovative therapies for patients with significant unmet need," said Dr. Bokhari. "We believe SkinJect has the potential to shift the treatment paradigm in Gorlin Syndrome by offering a localized, repeatable, non-surgical approach."

The meetings were conducted in collaboration with the Gorlin Syndrome Alliance. Medicus is advancing regulatory and clinical planning to support a registrational development pathway for SkinJect.

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