BRIEF — Clinuvel completes NDA submission for rare genetic blood disorder drug

Australian firm Clinuvel Pharmaceuticals has completed the submission of a New Drug Application (NDA) for its drug Scenesse (afamelanotide 16 mg) as the first proposed therapy for patients with the rare metabolic disorder erythropoietic protoporphyria (EPP) in the USA.

An approved NDA would allow Clinuvel to make Scenesse available to adult EPP patients in the USA as a first-line therapy.

The NDA includes data from five clinical trials in EPP and real-world experience from Europe, where it is already authorized for adult EPP patients.