PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) has asked the U.S. Food and Drug Administration to grant Rare Pediatric Disease Designation for SkinJect, its experimental treatment for patients with Gorlin Syndrome, a rare genetic disorder that can cause recurring basal cell carcinomas beginning in childhood.
The designation request marks the latest regulatory step for the company’s SkinJect program and could provide access to incentives intended to encourage development of treatments for rare pediatric diseases if the therapy ultimately wins FDA approval.
Medicus said the submission follows a previously filed Orphan Drug Designation application and an ongoing FDA review of a proposed registrational study design for the treatment.
Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, is a hereditary disorder linked to mutations in the Hedgehog signaling pathway. Patients can develop dozens, hundreds, or even more than 1,000 basal cell carcinomas over their lifetimes, with lesions often appearing during childhood or adolescence.
The company estimates the condition affects roughly 6,000 to 12,000 people in the United States.
SkinJect is a dissolvable microneedle array patch designed to deliver the chemotherapy drug doxorubicin directly into basal cell carcinoma lesions. The localized approach is intended to concentrate treatment at the tumor site while limiting systemic exposure.
“Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age,” said Dr. Raza Bokhari, Medicus’ executive chairman and chief executive officer. “We believe it has the potential to become an important non-surgical treatment option for these patients.”
According to Medicus, a recently completed Phase 2 study showed a 64% clinical clearance rate and a 55% complete response rate among evaluable patients in the highest-dose treatment arm, while maintaining what the company described as a favorable safety and tolerability profile.
If the FDA grants Rare Pediatric Disease Designation and the therapy later receives marketing approval, Medicus could become eligible for a Rare Pediatric Disease Priority Review Voucher. Such vouchers can be used to obtain expedited FDA review of another drug application or sold to another company.
The company said it will continue discussions with regulators as it advances SkinJect’s development for Gorlin Syndrome and other basal cell carcinoma indications.
Support the local news that supports Chester County. MyChesCo delivers reliable, fact-based reporting and essential community resources—free for everyone. If you value that, click here to become a patron today.