In de Business Spectator van vorige maand vond ik nog een artikel over de aanwijzing van de twee orphan drug statussen aan Clinuvel door de EMEA. Ook al ligt het al weer een tijdje achter ons, vind ik het toch nog interessant genoeg, om dit hier te plaatsen.
Contro
08/04/08 EMEA grants two orphan drug designations to Clinuvel
Clinuvel reaches milestone
TOP News
By a staff reporter
A drug used to treat people with UV-related skin diseases and cancer-related conditions, which has a side-effect of tanning the skin, has cleared a European regulatory hurdle.
Biopharmaceutical firm Clinuvel Phamaceutical's photo-protective drug CUV1647 has been granted two orphan medicinal product designations by the European Medicines Agency (EMEA).
Under the European Union definition, orphan medicinal products are intended for prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU.
The drug will offer preventative treatment for phototoxic reactions for sufferers of erythropoietic porphyria (EPP) disease and congenital erythropoietic porphyria (CEP), who are unable to expose themselves to light and UV.
EEP is a rare genetic disease with severe skin symptoms, and CEP causes severe skin blistering.
The drug has been tested on organ transplant patients who are fair-skinned and on prescribed immune-suppressant medication, who are more likely to develop skin cancer than the general population.
CUV1647 is also likely to attract attention for another reason: the implant, which looks like a grain of rice and is popped into the skin, stimulates the production of melanin, which turns the skin a darker colour.
While admitting that the tanning side-effect will likely boost the product's appeal, Clinuvel is at pains to describe CUV1647 as a pharmaceutical product, rather than a cosmetic one, saying regulators needed to be cautious of the likelihood of misuse.
"The drug has a very broad application," non-executive director Dr Roger Aston said. "We don't want to focus on the cosmetic side at this stage."
The drug would, however, provide a tan without the process of skin damage. "If it provides protection against skin ageing - that's great," he said.
Non-executive director Dr Roger Aston described the designations as a "milestone," saying it set the firm on the pathway to sales, which is "still a rarity in the biotech sector."
If the firm successfully completes the Phase III trial due in 2009, it will seek EMEA marketing authorisation for CUV1647 for the first indication; the final regulatory step before the start of sales in the European Union.
The Melbourne-based firm said it will seek orphan status in the US shortly. "We won't file until we're ready, but we're pretty close," said CEO Philippe Wolgen.
Clinuvel, which has offices in San Francisco and Zurich and listed in 2001, manufacturers the drug in the United States..
Intersuisse analyst Darren J Grubb described the designation as a "watershed' for the firm, adding that the industry would likely understand its significance more than the market.