Clinuvel Pharmaceuticals submits NDA for SCENESSE to Health Canada, aiming to treat rare genetic disorder

Clinuvel Pharmaceuticals has filed a New Drug Submission to Health Canada for its innovative therapy, SCENESSE, marking a potential breakthrough for Canadian patients suffering from erythropoietic protoporphyria (EPP), a rare genetic disorder that triggers severe pain and burns from light exposure. If approved, SCENESSE will be the first treatment for EPP in Canada, offering a lifeline to patients who currently have no other options. This development represents a significant step forward for Clinuvel in its mission to provide systemic photoprotection to patients worldwide.

Clinuvel’s Chief Scientific Officer, Dr. Dennis Wright, highlighted the importance of bringing SCENESSE to Canada, noting that formal approval will allow more patients access to the drug, which has been transformative in other countries. Canadian patients are currently accessing SCENESSE through the Special Access Program (SAP), but widespread availability hinges on Health Canada’s review process, which could take up to 300 days.

SCENESSE works by stimulating melanin production in the skin, protecting patients from both visible and ultraviolet light, which is vital for those suffering from EPP. Currently, two Canadian specialty centers are accredited to administer the treatment, with more planned following regulatory approval. The drug is already available in Europe, the United States, and other regions, where it has proven highly effective in preventing phototoxic reactions and improving patients’ quality of life.

Expert Opinion: Experts Call SCENESSE a Game Changer for EPP Treatment

Dermatology experts are praising SCENESSE for its revolutionary approach to treating EPP. Dr. Michael Ceresnie, a leading figure in photomedicine, expressed optimism about the drug’s potential in Canada, stating that it not only significantly reduces the severity of phototoxic reactions but also dramatically improves the lives of those affected by this debilitating condition. Ceresnie added that SCENESSE opens new avenues for treating other light-sensitive conditions in the future.

Clinuvel’s submission to Health Canada is seen as a pivotal moment for the company’s ongoing efforts to expand access to its therapies. With an estimated 280 Canadians affected by EPP, the approval of SCENESSE could transform lives across the country, allowing patients to finally live without constant fear of sunlight.

Looking Ahead: Expanding Treatment Access Across Canada

If approved, Clinuvel plans to rapidly expand treatment access by accrediting additional centers across the country. For now, Canadian EPP patients must rely on SAP for access to SCENESSE, but broader availability is on the horizon pending Health Canada’s review. This will be a crucial development for patients who have long suffered from the excruciating effects of EPP with no viable treatment options.