Saturday, May 24, 2014

Clinuvel Updates` Experts in light and skin

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Clinuvel’s management address patients’, shareholders’ questions

Over an extended period of time Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) has received a number of questions from patients, shareholders and the broader biotechnology community regarding the company’s program and the progress being made towards regulatory approval by the EMA (European Medicines Agency) of the company’s drug SCENESSE® (afamelanotide 16mg).
In this release, Clinuvel’s Chairman Stan McLiesh and CEO Dr Philippe Wolgen address those questions most frequently asked of the company.
How long will it take to complete the EMA review of SCENESSE®?
The EMA review is estimated to be completed between July and October 2014, yet further reviews by the Agency are possible.
Why has the review process taken so long?
The dossier we have filed is complex. Firstly, no other melanocortin has ever been submitted to European regulatory bodies.
Secondly, erythropoietic protoporphyria (EPP) is a complex disorder in that patients experience symptoms following light and sun exposure; their fear for the consequences of exposure causes patients to lead a withdrawn life and to deliberately avoid exposure. Patients’ aversion to light makes the interpretation of their clinical behaviour a challenge. In essence, this is the first time that evaluation of light exposure to skin is needed to be assessed in a pharmaceutical submission.
Third, the overwhelming demand for SCENESSE®, from experts and patients, needs to be assessed by the EMA and weighed in their final outcome. The entire evaluation of SCENESSE® requires careful evaluation of the drug’s profile over the past 20 years in general, and the past nine years in EPP patients in particular. Safety is a key issue, with the expected adverse events (commonly known as ‘side effects’, which every drug has) reviewed and an evaluation made of the pharmacovigilance (safety measures in relation to drug and its distribution) once the drug has entered the market.
Do you expect a positive response to your submission?
The decision is taken by the entire Committee for Human Medicinal Products (CHMP) which represents all members of the European Community. The Clinuvel team is optimistic that it will receive a positive response from the EMA which will enable us to provide access to the drug for European EPP patients.
What happens if there is a further delay in the approval of your application?
Naturally, there is a ‘plan B’. On the basis of the safety data generated over the past 20 years, there should not be any safety concerns. This is the first part of EMA’s equation of a risk-benefit analysis. The efficacy of SCENESSE® is subject to patients’ and physicians’ experiences; in this case the statistics are of supporting evidence. Clinical relevance of treatment can only be expressed and confirmed by patient groups and all global experts knowledgeable in the disease.
Should the drug be approved, what will it cost?
The drug is being priced in Italy and Switzerland and, depending on a number of parameters, the final price will be determined and revealed in due course. An important factor is the manufacturing and scalability of the product. However, in EPP Clinuvel will distribute to a relatively small number of patients.
When will you approach the FDA for approval in the USA?
Clinuvel’s teams have been in regular contact with the FDA during the program, as should be expected from a pharmaceutical company. It is increasingly apparent that EMA and FDA exchange information and thoughts. In this light an EMA approval will trigger Clinuvel’s New Drug Application in the US.
How long will this process take?
If a Fast Track Status is granted by the FDA for SCENESSE® treatment in EPP, the review process can be relatively swift.
Is there any way to access the drug now?
The drug has been available through special access schemes for EPP patients in Italy and Switzerland since 2010. Our clinical experiences are excellent, and the distribution of the drug is very much driven by the academic demand, which in turn is driven by patients’ choice to opt in for the treatment.
You’ve spoken many times about a dose for children in EPP. Has any progress been made here?
The ultimate objective of this management was to develop a treatment for children with EPP. The ordeal that parents and children go through in porphyria is unspeakable and really, at times, unimaginable. Clinuvel’s scientists and management had initially underestimated the burden of EPP on juvenile patients and their families, until we actually met with them and discussed their experiences.
The clinical tragedy lies in the fact that patients suffer in their normal development, since they do not understand why they feel out of place and in agony following light exposure. It is not sun that triggers the symptoms but any visible light. Since we live by light, plants and humans alike need it for a normal biological development. It usually takes 6-7 years before patients and parents link clinical symptoms to a light disorder, and even longer when physicians find a diagnostic pathway. In the meantime many of these children end up in the waiting room of clinical psychologists and psychiatrists to assess whether there is a somatic disorder at all.
Most of these adult patients have gone through a traumatic childhood and are still isolated while fearful of phototoxicity. Often burning ‘pain’ is being described as the main symptom, however the traditional understanding of pain is most likely incorrect: these patients do not respond to any analgesic, peripheral, central, NSAID, cyclooxygenase inhibitor or opioid. In our current medical dictionary we have failed to come up with another description of phototoxicity other than pain, however the terminology probably doesn’t do justice to these patients.
Clinuvel is working on a paediatric dose, but can really only accelerate this when EMA approval for adults is in sight.
When will the drug be available for vitiligo?
Vitiligo is in Phase II trials in Asia. An EPP approval will have a spill-over effect on the ability to progress SCENESSE® for vitiligo patients. A regulatory outcome will influence Clinuvel’s choices in vitiligo.
As stated, we are hopeful of a positive outcome, based on years of use of SCENESSE®, the strong and genuine clinical feedback and safety. This last aspect has always dominated and will dictate Clinuvel’s choices.
- End -
Investor relations contacts:
Australia: Clinuvel Pharmaceuticals Limited, T: +61 3 9660 4900
Europe: Clinuvel AG, T: +41 41 767 45 45
Media contact:
Lachlan Hay
Clinuvel AG
About SCENESSE® (afamelanotide 16mg)
SCENESSE® is a first-in-class therapeutic being developed by Clinuvel, with the generic name (or INN) afamelanotide. An analogue of α-MSH, afamelanotide is a linear peptide which activates eumelanin of the skin, the dark pigment which is known to provide photoprotective properties (offering skin protection against light and UV radiation). SCENESSE® is administered underneath the skin as a dissolvable implant approximately the size of a grain of rice. For more information on SCENESSE® go to
SCENESSE® is a registered trademark of Clinuvel Pharmaceuticals Ltd.
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe skin disorders. With its unique expertise in understanding the interaction of light and human skin, the company has identified three groups of patients with a clinical need for photoprotection and another group with a need for repigmentation. These patient groups range in size from 10,000 to 45 million. Clinuvel’s lead compound, SCENESSE® (afamelanotide), a first-in-class drug targeting erythropoietic protoporphyria (EPP), has completed Phase II and III trials in the US and Europe, and has been filed for review by the European Medicines Agency. Based in Melbourne, Australia, Clinuvel has operations in Europe, the US and Singapore.
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
  • "Remember....Research is the key to your cure!"

1 comment:

  1. Natural Herbs Clinic is a useful source of Treatment for Porphyria information, Porphyria Herbal Remedy is one of the best herbal remedies which use for the treatment of Porphyria. It treat the disease with out any side effects. Because it made with herbal ingredients.


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