CHMP Backs Afamelanotide, First Drug to Prevent Phototoxicity

Larry Hand


October 27, 2014

The European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for afamelanotide (Scenesse, Clinuvel), the first medicine aimed at preventing phototoxicity in adults with erythropoietic protoporphyria, a rare genetic disease that affects fewer than 10,000 people in the European Union.

And for the first time, patients were involved in the committee's decision.

Erythropoietic protoporphyria causes a stinging pain in affected individuals when exposed to sunlight, and if the exposure is prolonged, the pain can become incapacitating.

Afamelanotide works by stimulating production of a pigment, called eumelanin, that naturally protects the skin against phototoxic reactions.

The CHMP recommendation will go to the European Commission for a final decision.

The committee's path to a recommendation was somewhat unusual, and the recommendation falls under the category of exceptional circumstances. The applicant, Clinuvel Pharmaceuticals of Baar, Switzerland, is a small company and benefitted from free scientific advice from the committee because of an orphan disease designation.

As a result of difficulties in conducting placebo-controlled trials for this disease, because patients are unwilling to expose themselves to sunlight, the committee acted on data from more than 110 patients recruited into a global compassionate use program.

During the committee's proceedings, patients and healthcare providers involved in an expert group spoke about improvements in quality of life experienced by patients who took afamelanotide during the testing program.

The committee's recommendation comes with the condition that Clinuvel develop and implement a robust risk-management program. The company will establish a patient registry to monitor safety and efficacy of the drug if it is approved by the European Commission.


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