PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) said it submitted an Orphan Drug Designation application to the U.S. Food and Drug Administration for SkinJect (D-MNA) to treat basal cell carcinoma in patients with Gorlin Syndrome, a rare genetic disorder.
The company said Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is driven primarily by mutations in the PTCH1 gene and can lead to the development of multiple, recurrent skin cancers beginning in childhood.
Patients with the condition may develop dozens to more than 1,000 basal cell carcinomas over their lifetime, often requiring repeated surgical procedures, including excision or Mohs surgery, the company said.
Medicus said there are no FDA-approved therapies specifically for basal cell carcinoma in patients with Gorlin Syndrome, and existing systemic treatments, such as Hedgehog inhibitors, are limited by tolerability and recurrence after discontinuation.
SkinJect is a dissolvable microneedle array containing doxorubicin designed for direct intradermal delivery into tumor tissue, providing localized treatment with limited systemic exposure, according to the company.
The company said the therapy is intended to allow repeat treatment of multiple lesions over time through a minimally invasive approach.
SkinJect has been evaluated in Phase 1 and Phase 2 clinical studies in patients with basal cell carcinoma, where Medicus reported a favorable safety profile and no serious adverse events or dose-limiting toxicities.
The company also reported evidence of clinical clearance and complete histological responses in treated lesions in those studies.
Medicus said it estimates Gorlin Syndrome affects about 11,000 patients in the United States, below the 200,000-patient threshold required for orphan drug designation.
If granted, the designation could provide seven years of U.S. market exclusivity upon approval, as well as eligibility for tax credits, fee waivers, and increased regulatory interaction with the FDA, the company said.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News.