The results of the CUV017 study suggest that treatment with afamelanotide might help patients with erythropoietic protoporphyria (EPP) to lead more normal lives.
EPP is a genetic disease arising from a deficiency of the enzyme ferrochelatase, which leads to accumulation of protoporphyrin. Affected patients experience severe pain, swelling and scarring, usually of the hands and face, when exposed to light. They are often forced to remain indoors, severely affecting their quality of life.
The multicentre, randomised, double-blind trial included 91 patients with EPP, who received subcutaneous implants of 16mg afamelanotide or placebo alternately every 2 months for a year.
Afamelanotide is analogue of α-melanocyte-stimulating hormone (α-MSH), an endogenous peptide that triggers release of the photoprotective pigment eumelanin.
Patients receiving afamelanotide reported significantly fewer days affected by pain and a significant reduction in average pain severity compared with those given placebo (p=0.0023 and p=0.0017, respectively). As a result, they were able to tolerate significantly longer periods of sun exposure (p<0.0001).
No serious reactions to the drug occurred. Most adverse events were mild or moderate, with headache, nausea, flushing and gastrointestinal disturbance reported most often.
Full results of CUV017 will be presented at the 19th Congress of the European Academy of Dermatology and Venereology in Gothenburg on 9th October.
Afamelanotide is also being tested by developer Clinuvel for the prevention of skin cancer in organ transplant recipients and to treat polymorphic light eruption, among other indications.