Bangalore, Dec 21, 2009: People with the rare genetic disorder that makes them “intolerant to sunlight” which are further compounded by increasing ultraviolet (UV) radiation due to depleting ozone layer can look forward to effective treatment and even preventive medication. An Australian biotech company has developed a novel drug which shows promise as “UV vaccine.”
A 12-month global trial on over 100 patients with this rare disorder, called erythropoietic protporhyria (EPP) with afamelanotide in phase III trials has shown considerable promising treating these patients afflicted with light-sensitive skin disorders. The drug could also help in preventive treatment, said experts at the Melbourne-based company, Clinuvel, who developed this drug.
In fact, due to its promise as preventive medicine, it has been dubbed the “UV vaccine” by many experts. People with skin cancers too could benefit from treatment with this new drug.
“After years of development and managing uncertainty in drug development, we are approaching our objectives,” Clinuvel CEO, Dr Philippe Wolgen said in a global statement on December 21,2009. “Our primary goal is to develop a safe preventative drug with a positive impact on the lives of EPP patients. It is conceivable that others with severe skin diseases affected by light may also benefit from afamelanotide.”
Thousands of people around the world are afflicted with EPP, which leaves them in “intolerable pain,” with blistering and burns when exposed to normal levels of light and sunlight. The disease is incurable and affect patients for life. It is a rare genetic disorder.
Chemical-based sun screens don’t work with these patients and people afflicted with EPP have to cover up themselves fully whenever they step out into the sun drenched streets. Essentially some genetic disorders in these people do not block out visible light in the blue spectrum which causes toxic reactions in such patients.
This genetic disorder leads to an accumulation of protoporphyrin IX in the skin and an increased risk of incapacitating phototoxic reactions manifesting as pain and severe ulceration of the skin.
Clinuvel’s scientists have been working on developing afamelanotide for 19 years. Afamelanotide is a first-in-line therapeutic analogue of α-MSH. It is is a linear peptide which activates the skin to activate and produce eumelanin, the dark pigment which is known to have photo protective properties (providing skin protection against light and UV radiation).
Increased pigmentation of the skin appears a few days after administration of afamelanotide and lasts up to 60 days. Afamelanotide is administered underneath the skin as a dissolvable implant approximately the size of a grain of rice.
In 2008, afamelanotide was awarded orphan drug designation (ODD) in the treatment of EPP by the FDA and EMEA. This classification should pave the way for a fast track regulatory approval process.
Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg said: “The impact of this life-long disease is seen and reported in our trials by the adult patients who have been unable to lead a normal pain-free existence since early childhood. These first statistical analyses confirm the overwhelmingly positive anecdotal reports during the trial. The patients’ response to our novel therapy and the physicians’ assessment will play a significant role in the regulatory review process.”
The drug could reach the markets in late 2010 after some more trial results are evaluated by regulators in major markets.
| 
Comment on this article
