Medicus Pharma (NASDAQ:MDCX) said on Friday it has submitted an application to the US Food and Drug Administration seeking orphan drug designation for its SkinJect therapy to treat basal cell carcinoma in patients with Gorlin Syndrome, a rare genetic condition marked by recurring skin cancers.
The company said the filing expands its SkinJect program into an area of high unmet medical need, where treatment options are limited and often rely on repeated surgical procedures that can lead to cumulative physical and psychological burden.
Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is driven largely by mutations in the PTCH1 gene and can result in patients developing dozens to more than 1,000 tumors over their lifetime, often beginning in childhood. There are currently no FDA-approved therapies specifically for basal cell carcinoma linked to the condition, with care typically involving surgical excision or Mohs surgery, while systemic treatments can be constrained by tolerability and recurrence.
SkinJect is a dissolvable microneedle array containing doxorubicin designed to deliver the drug directly into tumor tissue.
According to Medicus, the localized approach enables high drug concentrations at the site of the lesion while minimizing systemic exposure, and could be used repeatedly to treat multiple tumors over time.
Early-stage clinical studies in broader basal cell carcinoma populations showed a favorable safety profile, with no serious adverse events or dose-limiting toxicities reported, and evidence of lesion clearance in some cases, the company said.
Medicus believes the therapy’s mechanism of combining direct tumor cell killing with localized immune activation may be suited for patients with Gorlin Syndrome, who require ongoing treatment for multiple lesions.
If granted, the designation would provide incentives including seven years of market exclusivity upon approval, as well as potential tax credits, fee waivers and increased regulatory support.