CLINUVEL files Canadian New Drug Submission for SCENESSE® in EPP

CLINUVEL has filed 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). If approved, 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 medications for use in Canada, assessing the safety and efficacy of the products in the proposed indication, as well as the drug’s quality. Following a formal dossier validation period, the HPFB may complete the review of a new drug candidate within 300 days.

Canadian Special Access Program ongoing

In 2023 CLINUVEL announced that the first Canadian EPP patient had received treatment with SCENESSE® under Canada’s Special Access Program (SAP). The SAP allows individual physicians to facilitate access to treatment for patients who have serious or life-threatening conditions and lack therapeutic alternatives. 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 support their treatment access.

Two Canadian Specialty Centers have been trained and accredited to treat EPP patients with SCENESSE®. Further potential Canadian treatment centres have been identified to enable prompt treatment access pending regulatory and pricing approvals. To date, CLINUVEL has trained and accredited 85 Specialty Centers across North America.

EPP affects approximately 1:140,000 individuals, with an estimated 280 EPP patients in Canada.

Commentary

“The SAP has provided an important bridge for Canadian patients to access treatment and helped us understand the Canadian therapeutic landscape,” CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said. “A formal authorisation will enable more Canadian patients to receive SCENESSE® and is a logical next step.

“The dossier submitted contains both data which led to the FDA’s approval, as well as long-term data collected during the follow up of EPP patients worldwide,” Dr Wright concluded.

SCENESSE® in EPP: systemic photoprotection

EPP is a rare genetic disorder which causes phototoxicity, debilitating reactions and burns following light exposure. CLINUVEL has spent nearly two decades developing SCENESSE® as the first treatment for EPP. The drug, administered as a controlled-release injectable implant every 60 days, stimulates the production of melanin in skin, protecting skin cells from visible and ultraviolet light (photoprotection) and acting as a strong antioxidant.

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

References

Ceresnie, M. S., et al. (2022). Association of quality of life measures with afamelanotide treatment in patients with erythropoietic protoporphyria and x-linked protoporphyria: A retrospective cohort study. Journal of the American Academy of Dermatology, S0190962222028729.

Elder, G., et al. (2013). The incidence of inherited porphyrias in Europe. Journal of Inherited Metabolic Disease, 36(5), 849–857.

Langendonk, J., et al. (2015). Afamelanotide for Erythropoietic Protoporphyria. The New England Journal of Medicine, 373(1), 48–59.

Wensink, D., Wagenmakers, M. A. E. M., & Langendonk, J. G. (2021). Afamelanotide for prevention of phototoxicity in erythropoietic protoporphyria. Expert Review of Clinical Pharmacology, 14(2), 151–160.

About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL (ASX: CUV; ADR LEVEL 1: CLVLY; Börse Frankfurt: UR9) is a global specialty pharmaceutical group focused on developing and commercialising treatments for patients with genetic, metabolic, systemic, and life-threatening, acute disorders, as well as healthcare solutions for specialised populations. As pioneers in photomedicine and the family of melanocortin peptides, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for systemic photoprotection, assisted DNA repair, repigmentation and acute or life-threatening conditions who lack alternatives.

CLINUVEL’s lead therapy, SCENESSE® (afamelanotide 16mg), is approved for commercial distribution in Europe, the USA, Israel, and Australia as the world’s first systemic photoprotective drug for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore, and the USA. For more information, please go to https://www.clinuvel.com.

Authorised for ASX release by the Board of Directors of CLINUVEL PHARMACEUTICALS LTD.

Head of Investor RelationsMr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD

Investor Enquirieshttps://www.clinuvel.com/investors/contact-us

Forward-Looking Statements

This release contains forward-looking statements, which reflect the current beliefs and expectations of CLINUVEL’s management. Please see CLINUVEL’s website for the full Forward-Looking Statements disclaimer.

Contact:Tel: +61 3 9660 4900Fax: +61 3 9660 4909Email: mail@clinuvel.comAustralia (Head Office), Level 22, 535 Bourke Street, Melbourne, Victoria, 3000, Australia