USA approves first therapy for rare genetic disorder

The US regulator has finally approved Scenesse (afamelanotide), a subcutaneous implant used to treat the ultra-rare disorder erythropoietic protoporphyria, in which exposure to sunlight causes pain.

The decision comes some five years after the European Medicines Agency (EMA) gave approval for the novel therapy. Reimbursement agencies have since given a mixed reaction to the option, with funding provided in Germany, but  not the UK.

Shares of the developer, the Melbourne-based biotech Clinuvel Pharmaceuticals (ASX: CUV), were boosted more than 60% following the announcement.