Clinuvel awarded orphan drug status by EU

Clinuvel Pharmaceuticals has received Orphan Medical Product (OMP) designation by the European Medicines Agency (EMEA) for its drug, afamelanotide, for the treatment of solar urticaria (SU).

OMP designation provides regulatory benefits to Clinuvel, such as access to assistance from the EMEA to facilitate the registration of afamelanotide for SU. This includes: scientific advice during the product development phase, fee reductions, access to the EMEA’s centralised approval procedures covering 27 member states and access to European Commission-funded research projects.

OMP designation also grants 10 years market exclusivity for afamelanotide for the treatment of SU in the EU, following a successful Marketing Authorisation Application.

Afamelanotide has already been granted OMP designation for the treatment of a different disorder, erythropoietic protoporphyria (EPP), in March 2008, but it now has similar designation for SU.

SU is a skin disorder marked by an acute allergic response following ultraviolet or Sun exposure. Symptoms can be systemic, such as anaphylaxis, breathing difficulty, nausea and headaches. Immediate localised reactions vary from characteristic ‘wheal formation’ and erupting flares on exposed skin sites to swelling of soft tissues.

SU patients typically avoid UV and visible light sources; to prevent outbreak of symptoms, they tend to live indoors in social isolation. It is estimated that three per 100,000 people suffer from SU worldwide.

Preliminary studies indicate that afamelanotide may be able to provide photoprotection to SU patients and prevent the onset of symptoms. Analysis of results from a Phase II clinical trial of afamelanotide in SU is anticipated shortly.

If the data warrant, Clinuvel will consider conducting further clinical trials to determine the ability of afamelanotide to satisfy this unmet medical need.

According to the EMEA, Orphan medicinal products are for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect nor more than five in 10,000 persons in the European Union. Pharmaceutical companies are unwilling to develop such medicinal products under normal market conditions, as the cost of bringing them to the market would not be recovered by the expected sales of the medicinal products without incentives.

“Today’s second orphan designation consolidates the position of afamelanotide as a systemic photoprotectant for a range of diseases," said Clinuvel’s CEO, Dr Philippe Wolgen.

"In 2006, we started the program on identified disease symptoms that are caused by UV and light. In using afamelanotide as a photoprotective drug in these indications, we established the beneficial use in the most severe diseases. It is rewarding to treat patients who have never been able to find medicinal answers to their diseases.”

Afamelanotide is an analogue of α-MSH, a peptide which activates the body’s natural ability to produce eumelanin, the dark pigment of the skin which is known to offer photoprotective properties, thus providing skin protection against UV radiation (UVR).

Increased pigmentation of the skin appears a few days after administration of afamelanotide and lasts up to two months. Afamelanotide is administered underneath the skin as a dissolvable implant approximately the size of a grain of rice.

Clinuvel is currently testing afamelanotide in five clinical indications with clinical trials at various levels: Sun/ultraviolet intolerance; Sun/ultraviolet poisoning; skin cancer in transplant patients; acute anaphylactic reaction to Sun/ultraviolet; phototoxicity following cancer treatment.

Phase I and II human clinical trials using afamelanotide have demonstrated that the drug is well tolerated and no significant safety concerns have been identified to date. Following successful conclusion of the development program, Clinuvel will work closely with global regulators to facilitate marketing approval of afamelanotide.