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news.Clinuvel Pharmaceuticals has received Italian Medicines Agency (AIFA) approval for the use and reimbursement of a drug under clinical investigation while it is in advanced regulatory review process worldwide.
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Patients diagnosed with the rare light and UV intolerance erythropoietic protoporphyria (EPP) will now be able to gain access to the photoprotective drug – known under the generic name Afamelanotide – on prescription. The decision by AIFA precedes approval of formal marketing authorisation by European or US drug regulatory agencies.
Afamelanotide is developed by Clinuvel and has been recognised for its innovative medicinal value by Italian regulators in the treatment of EPP. Patients diagnosed with EPP can now be prescribed the drug by a physician and the full cost will be reimbursed by the Italian National Health System (Sistema Sanitario Nazionale, SSN).
Clinuvel Pharma said that Afamelanotide works by activating pigment, melanin, in the skin providing a barrier between light and skin cells. To date, the photoprotective drug has been shown to be clinically safe and able to reduce the incidence and severity of reactions in EPP patients. Full results from a European Phase III study are expected shortly.
Hank Agersborg, chief scientific officer of Clinuvel Pharma, said : “AIFA has taken a leadership role in the treatment of absolute light and UV intolerance as seen in this kind of porphyria. We have seen a world-first in UV treatment: patients and physicians requesting regulators to allow access to the novel drug. Perhaps it is significant in today’s world that patients become more and more empowered in influencing drug development.”
Philippe Wolgen, CEO of Clinuvel Pharma, said: “Five years ago, Clinuvel made a decision to develop Afamelanotide specifically for patient populations who were most severely and acutely affected by UV and light. Today’s news comes as a welcome surprise but supports our choice to develop Afamelanotide in these categories of patients.
“Following the completion of the complex study of Afamelanotide in Italian EPP patients, we continued to support their treatment through a compassionate use program. Unfortunately, the indefinite free supply of Afamelanotide was not sustainable for Clinuvel as a small enterprise, and this approval by the Italian regulators not only makes the drug available to these trial patients, but also provides further incentive to the company to focus on orphan drug development.”