Medicus Pharma seeks FDA rare disease status
- Medicus Pharma (NASDAQ:MDCX) has submitted a Rare Pediatric Disease Designation (RPDD) request to the U.S. Food and Drug Administration (FDA) for SkinJect, its microneedle patch for treating basal cell carcinoma in Gorlin Syndrome patients.
- The company reported positive Phase 2 data, showing 64% clinical clearance and a 55% complete response rate for nodular basal cell carcinoma.
- Medicus stated that the RPDD and separate Orphan Drug Designation, if granted, could provide significant incentives including a Priority Review Voucher and seven years of U.S. market exclusivity.
Medicus Pharma (NASDAQ:MDCX) announced the submission of a Rare Pediatric Disease Designation request to the FDA for its SkinJect therapy.
The company is developing the doxorubicin-containing microneedle patch to provide a localized, non-surgical treatment for patients with Gorlin Syndrome, a rare genetic disorder.
Medicus noted that the program is supported by data from its SKNJCT-003 study and a registrational study design currently under FDA review.
Management stated that Orphan Drug Designation could offer benefits like an exemption from FDA application fees exceeding $5 million and seven years of market exclusivity.
If approved, the company stated that the RPDD could potentially yield a Priority Review Voucher, which may shorten the FDA review period for future applications.
Following the announcement, the Medicus share price was down at $0.33, and the company plans to continue its regulatory efforts to address this unmet medical need.
