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Clinuvel to file new drug with FDA

MELBOURNE, Australia and NEW YORK, July 18, 2016 (GLOBE NEWSWIRE) -- Clinuvel Pharmaceuticals (ASX:CUV) (OTC:CLVLY) (XETRA:UR9) today announced that the US Food and Drug Administration (FDA) has concluded an initial review of data from the use of its drug SCENESSE® (afamelanotide 16mg) in the rare genetic disorder erythropoietic protoporphyria (EPP). The FDA has deemed Clinuvel’s clinical data package satisfactory for submitting a New Drug Application (NDA) and a pre-NDA meeting is now being scheduled.

“We welcome the FDA’s review and conclusion that the initial clinical datasets are sufficient for filing a NDA for SCENESSE®,” Clinuvel’s CEO, Dr Philippe Wolgen said. “This is the culmination of more than a decade of work in EPP, a debilitating and very specific disorder for which there is no other treatment.”

“Having received Fast Track designation for SCENESSE®, Clinuvel’s teams are now working closely with FDA to commence the NDA filing on a rolling basis. A request will also be made for Priority Review, which shortens the review time from 10 to six months, to expedite access to this much needed drug for US EPP patients,” Dr Wolgen said.

SCENESSE® in EPP
EPP patients complain of burning under the skin, usually within minutes of exposure to bright lights, especially sunlight. The first sensation – phototoxicity – is the result of a chemical reaction taking place underneath patients’ skin which results in burns and damage of the tissues.

A first-in-class drug, SCENESSE® activates the pigment melanin in skin, providing a barrier which protects skin cells from light (photoprotection) while also acting as an anti-oxidant. SCENESSE® is delivered via a subcutaneous dissolving implant approximately the size of a grain of rice. Increased pigmentation of the skin appears after two days and lasts up to two months.

Five clinical trials of SCENESSE® in EPP showed the drug was well tolerated and could reduce the incidence and severity of EPP reactions to light (phototoxicity) in EPP. Trials also showed the drug could improve patients’ quality of life. Two phase III clinical trials, conducted in Europe and the USA, were published in the New England Journal of Medicine in 2015.1

SCENESSE® was approved by the European Medicines Agency in 2014 and is now commercially available for the prevention of phototoxicity in adult EPP patients in a number of European countries.2

FDA review
The FDA conducted a four month review of datasets from Clinuvel’s clinical trial program, deeming the data satisfactory for formal NDA submission. SCENESSE® has previously been granted both Fast Track and orphan drug designations by the FDA for the treatment of EPP, recognising the severity of the disorder and potential of SCENESSE® to provide clinical benefit where no other therapy exists.

An FDA-initiated EPP workshop will take place on October 24 to learn more about the disease, and the effectiveness and safety of the proposed treatment.3

“The FDA has shown a commitment to EPP patients and Clinuvel looks forward to expediting the availability of SCENESSE® in North America,” Dr Wolgen said. “This is a significant event for the patients for whom we work.”

1 Langendonk J et al (2015). Afamelanotide for Erythropoietic Protoporphyria. NEJM. 373(1):48-59.

2 SCENESSE® (afamelanotide 16mg) is approved in Europe as an orphan medicinal product for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with EPP. The innovative nature of the therapy in the orphan disorder, the lack of available scientific instruments to adequately measure the therapy, ethical considerations and the drug’s positive safety profile were some of the factors which led to the European marketing authorisation of SCENESSE®. Information on the product can be found on Clinuvel’s website at www.clinuvel.com.

3 FDA Scientific Workshop: 24 October 2016: http://www.fda.gov/drugs/newsevents/ucm501389.htm.

About erythropoietic protoporphyria (EPP)
Erythropoietic protoporphyria (EPP) is a rare genetic storage disorder that causes severe anaphylactic-like reactions to light (phototoxicity). Patients complain of burning under the skin, usually within minutes of exposure to bright lights, especially sunlight. The first sensation – phototoxicity – is the result of a chemical reaction taking place underneath patients’ skin which results in burns and damage of the tissues.

EPP symptoms can be acute, or delayed (subacute), most often expressed as generalised oedema, effusion in tissues and distortion of the skin. As little as a few minutes of light outdoors (even when it is overcast or transmitted through a window) or artificial light exposure may be sufficient to evoke EPP symptoms.

Phototoxicity is unresponsive to traditional pain and burn management techniques and patients can be incapacitated for days before reactions subside. Most patients withdraw from light exposure in order to manage their phototoxic symptoms.

About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd (ASX:CUV) (OTC:CLVLY) (XETRA:UR9) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe disorders. With its unique expertise in understanding the interaction of light and human skin, the Clinuvel has identified patient populations with a clinical need for photoprotection and for repigmentation. The worldwide prevalence of these patient groups range from 5,000 to 45 million. Clinuvel’s lead compound, SCENESSE® (afamelanotide 16mg), was approved by the European Commission in 2014 for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP).

Headquartered in Melbourne, Australia, Clinuvel has operations in Europe, Switzerland, the US and Singapore.

For more information go to http://www.clinuvel.com.

SCENESSE® is a registered trademark of Clinuvel Pharmaceuticals Ltd.

Forward-Looking Statements
This release may contain forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause Clinuvel’s actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: that the FDA may require additional studies beyond the studies planned for product candidates or may not provide regulatory clearances, including for SCENESSE®; that the FDA may not provide regulatory approval for any use of SCENESSE® or that the approval may be limited; that Clinuvel may never file an NDA for SCENESSE® regulatory approval in the US; that the Company may not be able to access adequate capital to advance its vitiligo programs; that the Company may not be able to retain its current pharmaceutical and biotechnology key personnel and knowhow for further development of its product candidates or may not reach favourable agreements with potential pricing and reimbursement agencies in Europe and the US.

Media enquiries Lachlan Hay, Clinuvel (UK) Ltd. +44 1372 860 765 Lachlan.Hay@clinuvel.com Nick Miles, Cabinet Privé de Conseils s.a. +41 22 321 4540 miles@cpc-pr.com Ted Agne, The Communications Strategy Group Inc. +1 718 631 3117 edagne@comstragroup.com Investor enquiries InvestorRelations@clinuvel.com

Source:Clinuvel Pharmaceuticals Limited