Clinuvel Files for FDA Approval of Treatment for Rare Metabolic Disorder

Rare Daily Staff

Clinuvel Pharmaceuticals said it has completed its submission for approval of Scenesse, an experimental therapy to treat erythropoietic protoporphyria, a rare metabolic disorder.

Erythropoietic protoporphyria causes severe reactions to light. Patients with the condition suffer physical burns and ulcers following exposure to light. This usually occurs within minutes of exposure to bright lights, especially sunlight. Exposure to artificial light may be sufficient to evoke symptoms. Phototoxicity is unresponsive to traditional pain and burn management techniques and patients can be incapacitated for days before reactions subside.

Scenesse won approval in Europe as an orphan drug for the prevention of phototoxicity in adult patients with EPP in 2014.

As part of its submission, Clinuvel included data and analyses from five clinical trials in EPP, data from Compassionate Use and Special Access programs, and data from the real-world experience of EPP patients receiving treatment in Europe. The data set consists of nearly 6,700 doses in more than 800 patients. A post-authorization pharmacovigilance plan to monitor patients in the United States is part of the submission.

The FDA granted Scenesse orphan drug designation for EPP in 2008. In July 2016 the FDA awarded Fast Track Designation allowing the company to make its final regulatory submission. Clinuvel has requested Priority Review and is awaiting a response from the FDA.

June 27, 2018