CLINUVEL Submits Canadian New Drug Application for SCENESSE®

MELBOURNE, Australia, October 1, 2024 (GLOBE NEWSWIRE) — CLINUVEL has submitted a New Drug Submission (NDS) to Health Canada seeking approval for its novel photoprotective therapy SCENESSE^® (Afamelanotide) for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). With permission SCENESSE^® would be the first treatment for Canadian EPP patients.

Health Canada review process

Health Canada's Health Products and Food Branch (HPFB) reviews and approves drugs for use in Canada, assessing the safety and effectiveness of the products in the proposed indication and the quality of the drug. After a formal dossier validation phase, the HPFB can complete the review of a new drug candidate within 300 days.

Canadian special access program underway

In 2023, CLINUVEL announced that the first Canadian EPP patient received treatment with SCENESSE^® as part of the Canadian Special Access Program (SAP). The SAP enables individual doctors to make it easier for patients with serious or life-threatening illnesses and a lack of treatment alternatives to access treatment. Patient treatment under the SAP continued without interruption prior to Health Canada's review of the NDS. All Canadian patients treated under the SAP have received insurance coverage to help them access treatment.

Two Canadian specialty centers have been trained and accredited to treat EPP patients with SCENESSE^®. Additional potential Canadian treatment centers have been identified to provide immediate access to treatment pending regulatory and pricing approvals. To date, CLINUVEL has trained and accredited 85 specialty centers across North America.

EPP affects approximately 1:140,000 people, and there are an estimated 280 EPP patients in Canada.

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“The SAP has provided an important bridge to access to treatment for Canadian patients and helped us understand the Canadian therapeutic landscape,” said Dr. Dennis Wright, Chief Scientific Officer of CLINUVEL. “Formal approval will allow more Canadian patients to receive SCENESSE^® and is a logical next step.

“The submitted dossier contains both data leading to FDA approval and long-term data collected during follow-up of EPP patients worldwide,” concluded Dr. Wright.

SCENE^® in EPP: systemic light protection

EPP is a rare genetic disorder that causes phototoxicity, debilitating reactions and burns after exposure to light. CLINUVEL has spent almost two decades developing SCENESSE^® as the first treatment for EPP. The drug, administered as a controlled-release injectable implant every 60 days, stimulates melanin production in the skin, protects skin cells from visible and ultraviolet light (photoprotection), and acts as a powerful antioxidant.

SCENESSE clinical and long-term post-marketing studies^® have shown that it can prevent and reduce the severity of phototoxic reactions and improve patients' quality of life. The drug has been approved for adults by the European Medicines Agency, the US Food and Drug Administration (FDA), and regulators in Australia and Israel. To date, over 16,000 cans of SCENESSE^® have been administered to EPP patients worldwide.

References

Ceresnie, MS, et al. (2022). Association between quality of life measures and afamelanotide treatment in patients with erythropoietic protoporphyria and x-linked protoporphyria: A retrospective cohort study. Journal of the American Academy of DermatologyS0190962222028729.

Elder, G., et al. (2013). The incidence of inherited porphyrias in Europe. Journal of Inherited Metabolic Diseases36(5), 849-857.

Langendonk, J., et al. (2015). Afamelanotide for erythropoietic protoporphyria. The New England Journal of Medicine373(1), 48-59.

Wensink, D., Wagenmakers, MAEM, & Langendonk, JG (2021). Afamelanotide for the prevention of phototoxicity in erythropoietic protoporphyria. Expert assessment of clinical pharmacology14(2), 151-160.

About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL (ASX: CUV; ADR LEVEL 1: CLVLY; Frankfurt Stock Exchange: UR9) is a global specialty pharmaceutical group focused on the development and commercialization of treatments for patients with genetic, metabolic, systemic and life-threatening acute diseases, as well as healthcare solutions for specialized populations. As a pioneer in photomedicine and the melanocortin peptide family, CLINUVEL's research and development has led to innovative treatments for patient populations with clinical needs for systemic photoprotection, assisted DNA repair, repigmentation and acute or life-threatening diseases Alternatives are lacking.

CLINUVEL's leading therapy, SCENESSE^® (Afamelanotide 16 mg) is approved for commercial distribution in Europe, the United States, Israel and Australia as the world's first systemic photoprotectant for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). CLINUVEL is headquartered in Melbourne, Australia, and has offices in Europe, Singapore and the USA. For more information, see

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Mr. Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD

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This press release contains forward-looking statements that reflect the current beliefs and expectations of CLINUVEL's management. Please see CLINUVEL's website for the full disclaimer of forward-looking statements.

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