PHILADELPHIA, PA — Medicus Pharma Ltd. (Nasdaq: MDCX) said its chief executive and leadership team met with U.S. lawmakers on Capitol Hill last month to seek support for regulatory and policy initiatives tied to its SkinJect® treatment for Gorlin syndrome, the company announced.
The meetings focused on advancing orphan drug designation, a registrational investigational new drug (IND) pathway for Gorlin syndrome patients, and support for a rare disease pediatric FDA voucher program.
Medicus said discussions included members of the House Energy and Commerce Committee and related subcommittees, including Chair Brett Guthrie, Rep. John Joyce, Rep. Diana Harshbarger and Rep. Gus Bilirakis.
The company conducted the outreach in collaboration with the Gorlin Syndrome Alliance to highlight the need for alternative treatments for patients with the rare genetic condition.
Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is caused primarily by mutations in the PTCH1 gene, which disrupt the Hedgehog signaling pathway and lead to recurring tumor growth.
Patients may develop dozens to more than 1,000 basal cell carcinomas over their lifetime, often beginning in childhood, requiring repeated surgical procedures.
There are no FDA-approved therapies specifically for basal cell carcinoma in Gorlin syndrome, and current treatment approaches rely largely on surgical removal or systemic therapies that may have tolerability limits and recurrence risks, according to the company.
Chief Executive Officer Dr. Raza Bokhari said the company is seeking to accelerate development and access to new treatment options.
“We believe SkinJect has the potential to shift the treatment paradigm … by offering a localized, repeatable, non-surgical approach,” Bokhari said.
Medicus said it is advancing clinical and regulatory planning for SkinJect, with the goal of generating data to support a registrational development pathway for patients with high disease burden.
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