EMA acknowledges Clinuvel's novel drug filing in Europe in 2011

EMA acknowledges Clinuvel's novel drug filing in Europe in 2011
Pre-Submission Meeting confirms in principle acceptability of SCENESSE® (afamelanotide) data package for evaluation of marketing authorisation application (MAA)
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it held a constructive Pre-Submission Meeting with the European Medicines Agency (EMA) on May 5. After reviewing the proposed content of the registration dossier for the novel drug afamelanotide (SCENESSE®), the EMA acknowledged that Clinuvel would meet all filing requirements. The EMA agreed with Clinuvel's tentative dossier submission period of the last quarter of 2011 for the orphan designated disease erythropoietic protoporphyria (EPP).
SCENESSE® is an injectable implant formulation of the first-in-class drug afamelanotide. The drug is administered every 60 days and mimics the body's natural ability to activate melanin in the skin. Melanin visibly darkens skin and provides biological protection to skin from ultraviolet and visible light, known medically as photoprotection.
In EPP, a rare disease causing absolute intolerance to light, SCENESSE® has been shown in clinical trials to provide photoprotection to patients, reducing the incidence and severity of painful phototoxic reactions caused by exposure of skin to visible light and sunlight.
The EMA Pre-Submission Meeting is the penultimate step of the European drug approval process prior to filing under the EMA's Centralised Procedure (CP). During this Meeting all relevant EMA departments have the opportunity to comment on the completeness of the documentation (Modules 1 to 5 of the Common Technical Document dossier) from an administrative perspective prior to formal evaluation of the marketing authorisation application (MAA) of a new drug.
During Clinuvel's Pre-Submission Meeting the EMA assessed whether the company had followed scientific advice provided in 2009 and asked Clinuvel to justify any deviations from this advice in its final dossier. Further guidance was also provided on optimising the submission procedure and technical sections of the afamelanotide dossier.
Clinuvel confirmed to the EMA that a Paediatric Investigational Plan (PIP), a development plan for the use of afamelanotide in children with EPP, will be submitted in May 2011. Finally, the EMA recommended that a Good Manufacturing Practice (GMP) audit be initiated by the company to facilitate the review process and confirmed that, since EPP is an orphan indication, no application fees will be payable. 
As part of coordinating the evaluation of Clinuvel's lead drug among the EMA member states, a rapporteur and co-rapporteur will be appointed within four weeks. Clinuvel intends to request a preliminary meeting with both these coordinating EMA officials prior to submitting its MAA and was encouraged to do so by the EMA. This meeting will cover scientific aspects of the reviewed application.
EMA's approval through the CP would allow Clinuvel to market afamelanotide under the brand name SCENESSE® in all 27 European Union member states as well as Norway, Iceland and Liechtenstein. At the completion of its EPP program, Clinuvel will have generated data on approximately 250 EPP patients in clinical trials.
"I am very pleased with the outcome of this meeting, as Clinuvel showed that it had followed the EMA's scientific advice and is compliant," Clinuvel's Chief Scientific Officer, Dr Hank Agersborg said. "Clinuvel's challenge now is to meet the agreed filing date later this year. The two confirmatory EPP studies CUV029 and CUV030 will be pivotal in the assessment of further efficacy and safety, the most important prerequisite for a successful submission."
"To arrive at this crucial stage within five years of starting the program in EPP is a truly exceptional achievement," added Clinuvel's CEO, Dr Philippe Wolgen. "Subject to positive results in the CUV029 and CUV030 trials, we will be able to submit efficacy data for 250 EPP patients and safety data for 450 additional photodermatoses patients evaluated across the clinical program."
- End -
For more information contact:                                                                               
Lachlan Hay, Head of Global Network and Communications, Clinuvel AG
T: +41 41 767 45 45, [email protected]
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd is a leading and innovative Australian company focused on the development of SCENESSE® (afamelanotide), its proprietary first-in-class photoprotective drug. Clinuvel has identified a number of groups of patients with a clinical need for photoprotection and one with a need for repigmentation therapy. Currently, Clinuvel is in its final stages to complete testing of SCENESSE® in Phase II and III trials in Australia, Europe and the United States. Clinuvel's ongoing focus is to demonstrate the safety and efficacy of SCENESSE®. Pending positive clinical results, Clinuvel aims to file SCENESSE® for its first market approval for the orphan indication porphyria (EPP).
For more information go to http://www.clinuvel.com. For more information on erythropoietic protoporphyria go to http://www.clinuvel.com/erythropoietic-protoporphyria
Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, SCENESSE® (afamelanotide) for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
·        actual results may and often will differ materially from these forward-looking statements;
·        no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved;
·        no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place
Level 14 / 190 Queen Street                                                 T +61 3 9660 4900                                            www.clinuvel.com
Melbourne, Victoria 3000                                                      F +61 3 9660 4999