Erythropoietic Protoporphyria (EPP)

Clinuvel's lead clinical indication for afamelanotide

Clinical and regulatory progress:

• European Phase III EPP trials completed and results due Q1 2010; see preliminary results here
• Confirmatory European Phase III EPP trials due to commence shortly
• US EPP trials awaiting FDA approval
• Orphan designation indication - read more

About Erythropoietic Protoporphyria (EPP)

Also known as EPP, erythropoietic protoporphyria is a rare inherited porphyrin metabolism disorder that affects between one in 200,000 and one in 750,000 people. This disorder causes a chemical known as protoporphyrin IX to accumulate in the skin. When the skin is exposed to the sun, these molecules undergo a chemical reaction that results in swelling, excruciating pain and scarring.

The pain is sometimes described as like having hot needles stuck into the skin. The lifelong pain experienced by these patients can be so severe that they require continuous treatment with analgesics to cope with the incessant pain. Typically, these patients become socially isolated because of the lack of an efficacious treatment and their need to continuously avoid sunlight.

Sun avoidance by remaining indoors or wearing sun protective clothing including cotton gloves and a wide brimmed hat is the first line in EPP management. Drugs such as B-carotene, cysteine and cimitedine have been used and because the disease is inherited, genetic counselling is recommended.

Since sun avoidance is recommended, patients lead lives where they are in the sun for very limited time. This can prevent normal social activities and the intense pain that is experience interferes with normal daily activities and can prevent adequate sleep.

It is hoped that with regular use, afamelanotide will be able to provide medicinal photoprotection to EPP patients, allowing them to lead more normal lives.

Clinical Results - EPP

In January 2009, Clinuvel announced interim results from its lead Phase III study of afamelanotide in patients diagnosed with EPP (CUV017). The data from the first 14 Swiss patients to complete the 12 month study period were analysed, showing afamelanotide was of clinical benefit in EPP. For more information, see the company's announcement.

In December 2009, Clinuvel announced preliminary results from its lead Phase III study of afamelanotide in patients diagnosed with EPP (CUV017). For more information, see the company's announcement.

Clinuvel anticipates releasing full results from CUV017 in Q1 of 2010.

Regulatory status

Afamelanotide has been granted Orphan Drug Designation by the EMA, FDA and Swissmedic for EPP.