CLINUVEL PHARMACEUTICALS LTD has filed a New Drug Submission (NDS) with Health Canada seeking approval for SCENESSE® (afamelanotide) for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). This submission marks a significant step toward providing the first approved treatment option for Canadian EPP patients.
Regulatory Review and Potential Approval
Health Canada's Health Products and Food Branch (HPFB) will review the submission, assessing the safety, efficacy, and quality of SCENESSE®. The review process may take up to 300 days following a formal dossier validation period. Approval would provide a much-needed treatment for a rare and debilitating condition.
Ongoing Special Access Program
Prior to this submission, Canadian EPP patients have been receiving SCENESSE® through Canada's Special Access Program (SAP). This program allows physicians to access treatments for patients with serious or life-threatening conditions when no alternatives are available. CLINUVEL reported that the first Canadian EPP patient received SCENESSE® under the SAP in 2023, and treatment has continued without interruption. All Canadian patients treated under the SAP have received insurance coverage.
Treatment Access and Infrastructure
To ensure timely access to treatment, CLINUVEL has trained and accredited two specialty centers in Canada to treat EPP patients with SCENESSE®. Additional potential treatment centers have been identified to further expand access, pending regulatory and pricing approvals. Currently, CLINUVEL has 85 Specialty Centers across North America trained and accredited.
Understanding Erythropoietic Protoporphyria (EPP)
EPP is a rare genetic disorder affecting approximately 1 in 140,000 individuals, with an estimated 280 patients in Canada. The condition causes extreme sensitivity to light, leading to phototoxic reactions and burns upon exposure. SCENESSE® is designed to address this by stimulating melanin production in the skin, providing photoprotection against visible and ultraviolet light, and acting as a strong antioxidant.
Clinical Evidence and Mechanism of Action
SCENESSE® (afamelanotide), administered as a controlled-release injectable implant every 60 days, has demonstrated its ability to prevent and reduce the severity of phototoxic reactions in EPP patients. Clinical and post-marketing studies have also shown improvements in patients' quality of life. The drug has already been approved for use in adults by the European Medicines Agency, the US Food and Drug Administration (FDA), and regulatory authorities in Australia and Israel. Over 16,000 doses of SCENESSE® have been administered to EPP patients worldwide.
Supporting Statements
CLINUVEL’s Chief Scientific Officer, Dr. Dennis Wright, stated that the SAP has been an important bridge for Canadian patients to access treatment and understand the Canadian therapeutic landscape. He added that the NDS dossier includes data that led to the FDA’s approval, as well as long-term data collected during the follow-up of EPP patients worldwide.
