Medicus Pharma (NASDAQ:MDCX) said on Monday its chief executive and senior leadership team met with US lawmakers on Capitol Hill to seek support for regulatory and policy measures aimed at advancing its SkinJect therapy for patients with Gorlin syndrome, a rare inherited condition linked to recurrent skin cancers.
The biotech company said the discussions focused on securing orphan drug designation for SkinJect for Gorlin syndrome, registrational investigational new drug approval for affected patients, and support for the rare disease pediatric FDA voucher program.
CEO Dr Raza Bokhari and the Medicus team met with senior members of the House Energy and Commerce Committee, including Chair Brett Guthrie, committee member John Joyce, Health Subcommittee Vice Chair Diana Harshbarger, and Congressional Rare Disease Caucus Co-Chair Gus Bilirakis.
The company said the outreach was coordinated with the Gorlin Syndrome Alliance and aimed at raising awareness of the need for new treatment options for patients with the condition, who often face lifelong recurrence of basal cell carcinomas.
Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is primarily caused by mutations in the PTCH1 gene, which disrupt the Hedgehog signaling pathway and can lead to early and repeated tumor formation.
Patients may develop dozens to more than 1,000 basal cell carcinomas over their lifetime, often starting in childhood, requiring repeated surgeries that can result in significant physical and psychological burden.
There are currently no US Food and Drug Administration-approved therapies specifically for basal cell carcinoma in Gorlin syndrome, with treatment largely relying on surgical excision or Mohs surgery.
Medicus said SkinJect is being developed as a localized, repeatable and non-surgical treatment option that could offer an alternative to repeated surgeries.
“Our engagement with policymakers reflects a clear alignment around accelerating access to innovative therapies for patients with significant unmet need,” Bokhari said in a statement.
The company said it is continuing regulatory and clinical planning to support a registrational development pathway for SkinJect, with the goal of generating data that could support expedited development for high-burden patient populations.