A novel Australian drug to protect patients who have light and sunlight intolerance will begin trials in the US next month.
The US Food and Drug Administration (FDA) has given permission to Melbourne's Clinuvel Pharmaceuticals to start a Phase II study of its drug, afamelanotide in up to 60 patients diagnosed with the rare light intolerance erythropoietic protoporphyria (EPP).
It is estimated that 3,000 Americans are known to be afflicted with EPP, a disease trademarked by intolerable pain as their skin burns and forms blisters when exposed to normal levels of light and sunlight. The disease is incurable.
As a result, patients with EPP spend most of lives indoors and lead a secluded life. The use of sunscreens does not help as they fail to block out visible light of the blue spectrum, which triggers toxic reactions in EPP.
The development of the world's first photoprotective light by Clinuvel over the past 10 years costs over A$70 million. The drug works by activating a barrier of pigmentation (melanin) between the light and the person's skin.
The implantable drug which has the size of a rice grain is able to induce and rise up skin pigmentation in fair skin patients who are less protected from UV effects.
Afamelanotide has been administered to over 500 patients in clinical trials in Europe and Australia. Its preliminary Phase II results show good safety and prevention of the first signs of EPP symptoms.
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, said Dr Philippe Wolgen, CEO of Clinuvel.
The 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.