FDA Gives Clinuvel Approval for Phase 2 Trial - Drug Discovery and Development

A novel Australian drug to protect patients who are intolerant of light and sunlight will begin trials in the US next month. The US Food and Drug Administration (FDA) has given clearance to Melbourne’s Clinuvel Pharmaceuticals to commence a Phase II study (CUV030) of its drug, afamelanotide, in up to 60 patients diagnosed with the rare light intolerance erythropoietic protoporphyria (EPP).

The six month study, to be conducted in medical centres in Alabama, California, New York, North Carolina, Texas and Utah, is the first therapeutic trial of afamelanotide in the US.

It’s estimated around 3,000 Americans are known to be afflicted with EPP, a disease characterized by “intolerable pain”: their skin blisters and burns when exposed to normal levels of light and sunlight. The disease is incurable and affects patients for life.

As a result, EPP patients spend most of their lives indoors and lead a secluded life. Sunscreens are of no use in this disease as they don’t block out visible light (in the blue spectrum), which is the cause of toxic reactions in EPP.

Clinuvel has spent over A$70 million the past 10 years developing afamelanotide as the world’s first photoprotective drug, which works to activate a barrier of pigmentation (melanin) between light and a person’s skin. The implantable drug, the size of a rice grain, is able to stimulate and increase skin pigmentation in fair- skin patients who are less protected from UV damage.

Afamelanotide has been administered to over 500 patients to date in clinical trials in Europe and Australia, with preliminary Phase III results showing good safety and first signs that EPP symptoms can be prevented.

“Clinuvel’s entry to the US to conduct a therapeutic trial is a major step event the company’s existence. In afamelanotide we are developing a unique drug to serve patients whose skin is most affected by ambient light.” Clinuvel’s CEO, Dr Philippe Wolgen said.

“Our principle goal is to develop a safe preventative therapeutic option for EPP patients, who are most severely affected by light and UV.

“This outcome also needs to provide relief to our loyal shareholders, as the company is now in the position to develop afamelanotide for an orphan indication in the US.”

Date: March 29, 2010
Source: Clinuvel Pharmaceuticals