Medicus Pharma Ltd. (MDCX) on Monday announced the submission of a Rare Pediatric Disease Designation (RPDD) request to the U.S. Food and Drug Administration for SkinJect for the treatment of basal cell carcinoma in patients with Gorlin Syndrome.
The RPDD submission follows the company's prior Orphan Drug Designation application and the SKNJCT-005 registrational study design, which remains under FDA review.
SkinJect is a dissolvable microneedle array that delivers doxorubicin (D-MNA) directly into nodular basal cell carcinoma lesions through intradermal administration.
Medicus Pharma recently completed a Phase 2 study that demonstrated positive results for SkinJect, with 64% clinical clearance and 55% histological clearance in patients with nodular basal cell carcinoma while maintaining a favourable safety and tolerability profile.
Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome (NBCCS), is a rare autosomal dominant genetic disorder caused by a mutation in the hedgehog signaling pathway. The condition results in the development of basal cell carcinoma, a common form of skin cancer.
According to the company, an estimated 6,000 to 12,000 people in the U.S. are affected by the disorder.
Given the significant unmet medical need associated with the disease, the company expects that the RPDD submission could support the regulatory strategy for SkinJect in Gorlin Syndrome and the development of a non-surgical treatment for recurrent basal cell carcinoma in patients with Gorlin syndrome who develop it in childhood.
If granted, RPDD could support the company's regulatory strategy for SkinJect in Gorlin syndrome, subject to FDA approval of a marketing application and eligibility to receive a priority review voucher.
Medicus shares closed Monday at $0.32, down 5.24%
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