Medicus Pharma announced the submission of a Rare Pediatric Disease Designation (RPDD) request to the FDA for SkinJect (doxorubicin microneedle array patch, D-MNA), targeting basal cell carcinoma in patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome.1 The submission follows the company's previously announced Orphan Drug Designation application and a registrational study design (SKNJCT-005) that remains under FDA review.2
Key Takeaways
- Medicus Pharma submitted a Rare Pediatric Disease Designation (RPDD) request to the FDA for SkinJect (D-MNA), an investigational doxorubicin-containing microneedle array patch, for basal cell carcinoma in patients with Gorlin Syndrome.
- Gorlin Syndrome is a rare autosomal dominant disorder driven by Hedgehog pathway mutations; patients may develop hundreds to more than 1,000 BCCs over a lifetime, with onset often in childhood.
- In a completed Phase 2 study, the 200 mcg D-MNA arm demonstrated 64% clinical clearance and 55% complete histological response in evaluable nodular BCC patients, with a favorable safety profile.
- SkinJect is designed for intradermal, lesion-directed doxorubicin delivery intended to maximize local drug concentration while minimizing systemic toxicity — a potential repeatable option as new lesions emerge.
- The RPDD submission follows a previously filed Orphan Drug Designation application and a registrational study design (SKNJCT-005) currently under FDA review.
- If RPDD is granted and SkinJect is ultimately approved, the program may be eligible for a Priority Review Voucher, potentially reducing FDA review time from ~10 months to ~6 months.
- No therapies are currently approved specifically for pediatric Gorlin Syndrome patients, representing a significant unmet need the company is targeting.
Gorlin Syndrome is a rare autosomal dominant disorder caused primarily by mutations in the Hedgehog signaling pathway. Patients are at risk of developing dozens to more than 1,000 BCCs during their lifetime, with lesions frequently appearing in childhood and adolescence. The condition affects an estimated 1 in 30,000 to 60,000 individuals worldwide, translating to approximately 6,000 to 12,000 patients in the United States.3
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SkinJect is a dissolvable microneedle array designed to deliver doxorubicin directly into nodular BCC lesions via intradermal administration. The mechanism is intended to achieve high drug concentrations at the tumor site while limiting systemic exposure. In the company's recently completed Phase 2 study, the 200 mcg D-MNA treatment arm demonstrated 64% clinical clearance and 55% complete histological response among evaluable nodular BCC patients, with a favorable safety and tolerability profile.
"Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age," said Dr. Raza Bokhari, Executive Chairman and CEO of Medicus Pharma. "SkinJect is designed to provide localized treatment directly to the lesion and our recently concluded Phase 2 study has shown positive results, with 64% clinical clearance and 55% complete response. We believe it has the potential to become an important non-surgical treatment option for these patients. The submission of our Rare Pediatric Disease Designation request represents another meaningful milestone in our efforts to advance SkinJect through the regulatory process and address a significant unmet medical need."
The Gorlin Syndrome development program was designed with input from clinical experts and the Gorlin Syndrome Alliance. Medicus has characterized the patient population as particularly well-suited for a repeatable, lesion-directed approach given the lifelong, recurring nature of the disease and a lack of approved therapies for pediatric patients.
If granted, RPDD could make SkinJect eligible for a Rare Pediatric Disease Priority Review Voucher upon future FDA approval — a transferable voucher that may reduce standard FDA review time for a New Drug Application from approximately 10 months to approximately 6 months. If Orphan Drug Designation is also granted and a marketing application is subsequently approved, SkinJect may be eligible for 7 years of US market exclusivity for the designated indication, along with certain FDA fee waivers.
Medicus said it intends to continue engaging with the FDA on the clinical development pathway for SkinJect in Gorlin Syndrome while advancing broader development opportunities in basal cell carcinoma.
References
- Medicus Pharma announces US Food and Drug Administration (FDA) submission of rare pediatric disease designation request for SkinJect® in Gorlin Syndrome. News release. Medicus Pharma. June 3, 2026. Accessed June 15, 2026. https://www.globenewswire.com/news-release/2026/06/15/3311700/0/en/medicus-pharma-announces-u-s-food-and-drug-administration-fda-submission-of-rare-pediatric-disease-designation-request-for-skinject-in-gorlin-syndrome.html
- Medicus Pharma advances SkinJect into registrational development for Gorlin syndrome. News release. Medicus Pharma. June 3, 2026. Accessed June 3, 2026. https://tinyurl.com/ycy7ruw8
- Murgia G, Valtellini L, Denaro N, et al. Gorlin syndrome-associated basal cell carcinomas treated with vismodegib or sonidegib: a retrospective study. Cancers (Basel). 2024;16(12):2166. Published 2024 Jun 7. doi:10.3390/cancers16122166
