Medicus Pharma (NASDAQ:MDCX), a clinical-stage biopharmaceutical company, announced today that it has submitted an application for Orphan Drug Designation (ODD) to the U.S. Food and Drug Administration (FDA) for its flagship candidate, SkinJect (D-MNA).
The application specifically targets the treatment of basal cell carcinoma (BCC) in patients suffering from Gorlin Syndrome, a rare genetic disorder characterized by the development of numerous skin cancers throughout a patient's life.
SkinJect is a patented, dissolvable microneedle array (D-MNA) that delivers a concentrated dose of doxorubicin, a well-established chemotherapeutic agent, directly into the tumor.
Unlike traditional systemic chemotherapy, SkinJect provides localized delivery that targets the lesion while minimizing exposure to healthy surrounding tissue.
The submission is supported by encouraging data from a Phase 1/2 clinical trial.
The study reported high rates of lesion clearance and "histologic complete responses," suggesting that the microneedle technology can effectively eliminate BCC tumors without the scarring or recovery time associated with surgical excision—the current standard of care.
Gorlin Syndrome affects approximately 11,000 people in the United States.
These patients often face a lifetime of repetitive, disfiguring surgeries to remove hundreds of BCC lesions.
Medicus Pharma highlights that SkinJect’s localized, repeat-use profile could represent a paradigm shift in managing this chronic condition.
If granted, Orphan Drug Designation would provide Medicus Pharma with several key benefits, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, and a waiver of FDA user fees.
This regulatory path is designed to incentivize the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S.