Medicus Pharma Announces U.S. Food and Drug Administration (FDA) Submission of Rare Pediatric Disease Designation Request for SkinJect® in Gorlin Syndrome
Rhea-AI Summary
Medicus Pharma (NASDAQ:MDCX) submitted a Rare Pediatric Disease Designation (RPDD) request to the FDA for SkinJect, its doxorubicin microneedle patch, to treat basal cell carcinoma in Gorlin Syndrome patients.
Medicus highlights Phase 2 data showing 64% clinical clearance and 55% complete response in nodular BCC with a favorable safety profile, and outlines potential RPDD and Orphan Drug incentives, including a future Priority Review Voucher and U.S. market exclusivity.
Positive
- RPDD request submitted to FDA for SkinJect in Gorlin Syndrome
- Phase 2 200µg arm showed 64% clinical clearance in nodular BCC
- Phase 2 200µg arm showed 55% histological complete response
- Phase 2 results reported with favorable safety and tolerability profile
- Potential eligibility for Priority Review Voucher if RPDD and future approval obtained
- Potential seven years of U.S. market exclusivity with future Orphan Drug approval
Negative
- Rare Pediatric Disease and Orphan Drug designations are not yet granted
- Registrational study design SKNJCT-005 remains under FDA review
News Market Reaction – MDCX
In the Jun 15 session, MDCX declined 5.24%, reflecting a notable negative market reaction. Argus tracked a peak move of +9.0% during that session. Our momentum scanner triggered 7 alerts that day, indicating moderate trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Jun 11 | Teverelix Phase 2a | Positive | +4.0% | IND for PRECISION-E2 Phase 2a Teverelix endometriosis trial in the UAE. |
| Jun 08 | EU trial submission | Positive | -3.1% | European submission to support Phase 2b Teverelix trial in high-risk prostate cancer. |
| Jun 03 | SkinJect registrational move | Positive | +7.0% | Protocol SKNJCT-005 submitted to FDA, moving SkinJect into Phase 2b for Gorlin Syndrome. |
| May 28 | Non-dilutive financing | Positive | +6.7% | Up to $22M structured facility, boosting cash and extending operating runway. |
| May 14 | Q1 2026 earnings | Negative | -10.7% | Reported loss, low cash, ATM use and going‑concern doubt in quarterly results. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Positive clinical, regulatory, and financing news has more often led to aligned price moves, with one recent divergence on favorable Teverelix progress.
Over recent months, Medicus has combined clinical progress with balance-sheet moves. A May 28 non-dilutive facility of up to $22 million supported a pro forma cash runway beyond 24 months. SkinJect® advanced into Phase 2b registrational development for Gorlin Syndrome on Jun 3, using Phase 2 data showing 64% clinical and 55% histological clearance. Teverelix® programs advanced in prostate cancer and endometriosis across multiple regions. This RPDD submission adds another regulatory layer tied specifically to SkinJect’s Gorlin Syndrome strategy.
Key Terms
rare pediatric disease designation regulatory
orphan drug designation regulatory
phase 2 medical
phase 2b medical
priority review voucher regulatory
new drug application regulatory
investigational new drug regulatory
basal cell carcinoma medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
Submission Supports Development of a Potential Non-Surgical Treatment for a Rare Genetic Disorder that Causes Recurrent Basal Cell Carcinomas Beginning in Childhood
PHILADELPHIA, June 15, 2026 (GLOBE NEWSWIRE) -- Medicus Pharma Ltd. (NASDAQ: MDCX) (“Medicus” or the “Company”), a biotech/life sciences company focused on advancing the clinical development programs of novel and potentially disruptive therapeutic assets, today announced the submission of a Rare Pediatric Disease Designation (“RPDD”) request to the U.S. Food and Drug Administration (“FDA”) for SkinJect®, the Company’s investigational doxorubicin-containing microneedle array patch (D-MNA), for the treatment of basal cell carcinoma (“BCC”) in patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome.
The RPDD submission was made pursuant to Section 529 of the Federal Food, Drug, and Cosmetic Act and follows the Company’s previously announced Orphan Drug Designation (“ODD”) application (DRU-2026-11578), and Registrational study design (SKNJCT-005) which remains under FDA review. The design of the Company’s Gorlin Syndrome development program has been informed through engagement with clinical experts and the Gorlin Syndrome Alliance, a patient advocacy group focused on the needs and priorities of this unique patient population.
“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 CEO, “SkinJect® is designed to provide localized treatment directly to the lesion and our recently concluded Phase 2 study has shown positive results, with
Gorlin Syndrome is a rare autosomal dominant genetic disorder caused primarily by mutations affecting the Hedgehog signaling pathway. Patients may develop dozens, hundreds, or even more than one thousand basal cell carcinomas during their lifetime. These lesions often appear during early childhood and adolescence and continue to develop throughout life. The condition is estimated to affect approximately 1 in 30,000 to 60,000 individuals worldwide, corresponding to an estimated U.S. patient population of approximately 6,000 to 12,000 individuals and a substantially larger addressable population across major global markets.
SkinJect® is a proprietary dissolvable microneedle array designed to deliver doxorubicin (D-MNA) directly into nodular basal cell carcinoma lesions through localized intradermal administration. The technology is intended to achieve high drug concentrations at the tumor site while minimizing systemic exposure and associated toxicities.
In the Company’s recently completed Phase 2 study, the 200ug D-MNA treatment arm demonstrated the strongest rates of clinical and histological clearance of
The Company believes the clinical profile observed to date supports the continued evaluation of SkinJect® as a repeatable, lesion-directed therapy for patients who may develop numerous BCC lesions over the course of their lifetime.
If granted, Rare Pediatric Disease Designation would further support the Company’s regulatory strategy for SkinJect® in Gorlin Syndrome. Subject to compliance with applicable statutory requirements and future FDA approval of a marketing application for the designated indication, the program may provide eligibility for a Rare Pediatric Disease Priority Review Voucher. Such a voucher may be used to obtain priority review of a future marketing application or transferred to another company.
A Priority Review Voucher, if awarded and utilized, may reduce FDA review time for a future New Drug Application from approximately ten months under standard review to approximately six months under priority review, potentially accelerating patient access to innovative therapies and shortening the time required to bring new treatments to market.
In addition, if Orphan Drug Designation is granted and a future marketing application is approved, SkinJect® may become eligible for seven years of U.S. market exclusivity for the designated indication, waiver of certain FDA application fees, and additional regulatory incentives intended to encourage development of therapies for rare diseases.
The Company believes that Gorlin Syndrome represents a compelling development opportunity for SkinJect® given the significant unmet medical need, the recurring nature of the disease, the lack of approved therapies for pediatric patients, and the potential for a lesion-directed, non-surgical treatment approach that may reduce dependence on repeated surgical procedures. The Company further believes SkinJect® may be uniquely positioned to address both pediatric and adult manifestations of Gorlin Syndrome through a repeatable, lesion-directed treatment approach designed to manage newly emerging lesions throughout the course of the disease.
Medicus intends to continue engaging with the FDA regarding the clinical development pathway for SkinJect® in Gorlin Syndrome while advancing broader development opportunities in basal cell carcinoma.
For further information contact:
Carolyn Bonner, President and Chief Financial Officer
(610) 636-0184
cbonner@medicuspharma.com
Anna Baran-Djokovic, SVP Investor Relations
(305) 615-9162
adjokovic@medicuspharma.com
About Medicus Pharma Ltd.
Medicus Pharma Ltd. (Nasdaq: MDCX) is a precision-guided biotech/life sciences company focused on accelerating the clinical development programs of novel and potentially disruptive therapeutic assets. The Company is actively engaged in multiple countries across three continents.
The Company’s current key therapeutic assets are:
SkinJect®, a novel localized immuno-oncology precision product focused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) and Gorlin Syndrome, a rare autosomal dominant disease also called nevoid BCC syndrome, collectively representing a ~
Teverelix®, a next-generation GnRH antagonist, is a first-in-market product for cardiovascular high-risk advanced prostate cancer patients and patients with acute urinary retention relapse (AURr) episodes due to enlarged prostate, collectively representing a ~
Cautionary Notice on Forward-Looking Statements
Certain information in this news release constitutes "forward-looking information" under applicable securities laws. "Forward-looking information" is defined as disclosure regarding possible events, conditions or financial performance that is based on assumptions about future economic conditions and courses of action and includes, without limitation, statements regarding the collaboration with Gorlin Syndrome Alliance (GSA) including the potential benefits thereof for GSA, those suffering with Gorlin Syndrome and Medicus (including as it relates to the development of SkinJect®), ability to be approved for the Registrational IND Program to enable those suffering with Gorlin Syndrome to access SkinJect® under physician-supervised treatment protocols, the submission of Protocol SKNJCT-005 and the design, timing, conduct and results of the SKNJCT-005 Phase 2b registrational study, including whether the resulting data will be sufficient to support a future New Drug Application for SkinJect® in Gorlin Syndrome, the potential for SkinJect® to become the first FDA-approved lesion-directed therapy for patients with Gorlin Syndrome and a new standard of care for this patient population; and the potential issuance and benefits of a Rare Pediatric Disease Priority Review Voucher if the Company’s submission request is approved, Orphan drug designation for SkinJect®, the development of Teverelix® and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of Teverelix® for AURr, cardiovascular high-risk advanced prostate cancer, women’s health indications like endometriosis, and the potential market opportunities related thereto, the development, advancement and commercialization of SkinJect® through SKNJCT-003 and SKNJCT-004, and the potential market opportunities related thereto, the Company’s expectations regarding reported efficacy findings of SkinJect®. Forward-looking statements are often but not always, identified by the use of such terms as "may", “on track”, “aim”, "might", "will", "will likely result", “could,” “designed,” "would", "should", "estimate", "plan", "project", "forecast", "intend", "expect", "anticipate", "believe", "seek", "continue", "target", “potential” or the negative and/or inverse of such terms or other similar expressions. These statements involve known and unknown risks, uncertainties and other factors, which may cause actual results, performance or achievements to differ materially from those expressed or implied by such statements, including those risk factors described in the Company's annual report on form 10-K for the year ended December 31, 2025, and in the Company's other public filings on EDGAR and SEDAR+, which may impact, among other things, the trading price and liquidity of the Company's common shares. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement and reflect our expectations as of the date hereof and thus are subject to change thereafter. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Readers are further cautioned not to place undue reliance on forward-looking statements as there can be no assurance that the plans, intentions or expectations upon which they are placed will occur. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated.
