- Australian drugmaker Clinuvel on Tuesday won U.S. approval for a drug designed to treat a rare skin disease that causes painful sensitivity to light, capping more than a decade of development by the Melbourne-based company.
- Shares in Clinuvel jumped 60% on Australia's principal stock exchange following news of the Food and Drug Administration OK, valuing the company at more than $2 billion.
- Scenesse, as the drug will be sold, is cleared to treat people suffering from erythropoietic protoporphyria, an inherited disorder that results in intense skin pain and skin thickening.
Clinuvel's Scenesse (afamelanotide) is the first drug cleared in the U.S. to help people with erythropoietic protoporphyria increase their light exposure.
Approval by the FDA comes five years after the drug was OK'd in Europe, and 13 years after clinical testing began.
Regulatory decisions were based on two parallel group studies that gave trial participants either Scenesse or a placebo form of the implant every two months.
Both measured the total number of hours spent either outdoors, or in direct sunlight, on days when patients reported no pain. People who received Scenesse were able to be exposed to light for longer without pain, study results showed, although in both cases the amount of time remained quite limited.
In one, for example, Scenesse-treated participants spent a median of 64 hours in direct sunlight on days with no pain over the course of six months.
Erythropoietic protoporphyria is an autosomal recessive disorder that stems from decreased activity of an enzyme called ferrochelatase. That deficiency sets off a chain reaction leading to accumulation in the body of protoporphyrin IX, which can react with light reaching the skin.
Scenesse is an analog of a human hormone that stimulates melanin-producing skin cells. By binding to the melanocortin 1 receptor, Clinuvel's drug increases the production of photoprotective eumelanin in the skin.
The FDA granted Scenesse approval under Priority Review.