Singapore, December 17, 2009: Clinuvel Pharmaceuticals Limited, an innovative Australian company focused on the development of afamelanotide announced that its photoprotective drug afamelanotide has been granted an orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the management of solar urticaria (SU). This is the second ODD granted to afamelanotide by the FDA since its awarded afamelanotide its ODD status for the treatment of erythropoietic porphyrias, a genetic haematological disorder.

 

Dr Philippe Wolgen, CEO, Clinuvel said, “This additional FDA award for afamelanotide boosts our confidence in the choices we have made for this development program, for which we identified that our drug would provide the optimum medical utility and benefit for patients affected by UV and light. After more than a decade of development, a critical factor to the commercial success of the drug will hinge on market protection and exclusivity.”

 

In July 2009, Clinuvel announced positive results from a pilot Phase II study of SU, showing SU patients’ tolerance to light (UV and other wavelengths) increased following the administration of afamelanotide. Afamelanotide was granted orphan medicinal product status by the European Medicines Agency for SU in June 2009. Clinuvel is preparing final Phase III trials in SU to start in the spring in the northern hemisphere in 2010.

 

The FDA’s orphan drug designation is reserved for new drugs or therapies being developed to treat rare diseases or conditions that affect smaller populations in the United States. The orphan drug designation allows for an accelerated review process by the FDA, seven-year market exclusivity in the United States upon obtaining marketing authorisation, tax benefits, and exemption from user fees.

 

SU is a skin disorder characterised by an acute mast cell (allergic) response to the photo antigen UV or light. Symptoms can be systemic, such as anaphylaxis, breathing difficulty, nausea and headaches. Immediate localised reactions vary from typical ‘wheal formation’ and erupting flares on exposed skin sites to swelling of soft tissues. SU patients are forced to avoid outdoors existence in the spring and summer each year to prevent acute episodes; these patients tend to live in social isolation. From the known patient registers, it is estimated that more than 5,000 people are diagnosed with SU worldwide.