Afamelanotide Beneficial In Erythropoietic Protoporphyria

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An α-melanocyte-stimulating hormone analog, afamelanotide, is associated with reduced pain and improved quality of life in patients with erythropoietic protoporphyria.
An α-melanocyte-stimulating hormone analog, afamelanotide, is associated with reduced pain and improved quality of life in patients with erythropoietic protoporphyria.

HealthDay News -- An α-melanocyte-stimulating hormone analog, afamelanotide, is associated with reduced pain and improved quality of life in patients with erythropoietic protoporphyria, according to new research published in the New England Journal of Medicine.

Janneke G. Langendonk, MD, PhD, from Erasmus Medical Center in Rotterdam, Netherlands, and colleagues examined the safety and efficacy of afamelanotide for erythropoietic protoporphyria. Seventy-four patients from the European Union and 94 from the United States were randomized in a 1:1 ratio to receive a subcutaneous implant containing afamelanotide or placebo every 60 days (five implants in the European Union study; three in the U.S. study).

The researchers found that the duration of direct exposure to sunlight without pain after six months was longer in the afamelanotide versus placebo group in the U.S. study (median, 69.4 versus 40.8 hours; P = 0.04). The duration of pain-free time after nine months was longer in the afamelanotide versus placebo group in the European Union study (median, 6.0 versus 0.8 hours; P = 0.005); there was also a lower number of phototoxic reactions in the afamelanotide group (77 versus 146; P = 0.04). Quality of life improved with afamelanotide therapy in both studies.

"Afamelanotide had an acceptable side-effect and adverse-event profile and was associated with an increased duration of sun exposure without pain and improved quality of life in patients with erythropoietic protoporphyria," the authors write.

Several authors disclosed financial ties to pharmaceutical companies, including Clinuvel Pharmaceuticals, which manufactures afamelanotide and partially funded the study.

Reference

1. L Janneke , et al. N. Engl. J. Med. 2015; doi:10.1056/NEJMoa1411481.

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