Dutch See First European Launch Of Clinuvel's Scenesse For Phototoxicity In EPP
Executive Summary
Clinuvel is beginning the commercial launch in Europe of its orphan drug Scenesse for patients with erythropoietic protoporphyria, a rare condition that causes severe anaphylactic reactions and burns when the skin is exposed to sunlight.
Australian firm Clinuvel Pharmaceuticals Ltd. has announced the first commercial launch in Europe of its orphan drug Scenesse (afamelanotide) for the rare genetic disorder erythropoietic protoporphyria (EPP).
Patients in the Netherlands are this week beginning treatment with Scenesse, which received EU approval in 2014 for the prevention of phototoxicity (anaphylactic reactions and burns) in adults with EPP. The first treatments in Austria and Germany are expected to begin in July.
Clinuvel said that in the Netherlands, which has one of the largest known adult EPP patient populations in Europe, Scenesse has been accepted as a specialty hospital product by the health authorities and "holds the distinction of being the first ever approved standard of care for EPP," enabling reimbursement for all adult patients in the country.
The company is planning to make the product available in countries where there are known EPP populations, focusing initially on countries where it has already been used in clinical trials or compassionate-use programs.
"We have identified a small number of European countries who can be considered first adopters in the rare disease space and have recognized the need to treat EPP patients during conditions when light intensity increases," said Clinuvel CEO Philippe Wolgen.
In Europe the product is being made available only through specialist porphyria expert centers, for which Clinuvel provides training and accreditation. In Germany, for example, it is expected that in the first year, three centers in Berlin, Chemnitz and Düsseldorf will be able to prescribe the treatment.
As to the question of cost and patient access to the drug, the company says it is in the process of establishing a "uniform commercial price" for Scenesse across Europe, although it has not given any details on the actual price level.
Discussions on access are currently underway with authorities in a total of nine countries, with more submissions expected during the course of this year, the company said. 13 German insurance companies have agreed to make Scenesse available, and insurers in Austria have "facilitated access to Scenesse" for EPP patients, it added.
Company chairman Stan McLiesh said he was "proud that our teams have successfully navigated the onerous European and national systems to arrive at this point, and I look forward to broadening the availability of Scenesse in the coming months."
Safety Monitoring
EPP is a rare inherited metabolic disorder in which the chemical protoporphyrin accumulates in the skin. Exposure to the sun and ultraviolet radiation causes a chemical reaction that results in swelling, severe and intolerable pain and scarring, a condition known as phototoxicity. Afamelanotide, a chemical analogue of α-MSH which activates melanin, mimics the skin’s natural protection against UV rays and sunlight. It is delivered as a subcutaneous implant about the size of a rice grain. Increased pigmentation of the skin appears after two days and lasts up to two months, according to Clinuvel.
Scenesse has an "exceptional circumstances" marketing authorization, meaning that comprehensive data on the product may never become available or it would be unethical to gather those data. It was also unusual in that it involved a high degree of patient input during the evaluation process at the EMA.
).Patients treated with the drug are being encouraged to take part in a post-approval, non-interventional treatment protocol as part of a disease registry that is required by the European Medicines Agency. As part of the marketing authorization, Clinuvel agreed with the EMA to implement a risk management plan (RMP) comprising a Post-Authorization Safety Study (PASS) as well as a Retrospective Chart Review (RCR) to follow up those patients who do not consent to taking part in the PASS.
"The long term success of Scenesse lies in the safety profile of the product for EPP patients and, hence, our and regulators’ focus on pharmacovigilance," McLiesh noted.
Availability To Date
Clinical trials of Scenesse were conducted from 2006 to 2013, during which time it was evaluated in 352 EPP patients, according to Clinuvel. After the trial program ended, about 130 patients in seven European countries (Austria, France, Germany, Italy, the Netherlands, Sweden and Switzerland) and Australia received it free of charge. The company said that in total, 601 implant injections were given to patients under compassionate-use programs, and both these and the development program were supported entirely by equity funding.
The company said that Scenesse had also been made available pre-approval under a subsidized access scheme in Italy and Switzerland from 2010 and 2012 respectively, allowing 115 EPP patients to be treated, but that it was "no longer in the position to provide access to Scenesse on a subsidized basis."
It added that the next step for Scenesse was to develop it for use in children. It is also being investigated in a range of light and UV-related skin disorders and as a repigmentation therapy for vitiligo in combination with narrowband UVB phototherapy.