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US FDA pathway opens to SCENESSE® following Fast Track designation for treatment of EPP

  • FDA awards Fast Track Designation¹ to expedite review of SCENESSE® in EPP
  • NDA filing allowed on a rolling basis
  • FDA hosting a Scientific Workshop² on EPP to learn more about disease and drug treatment

MELBOURNE, Australia and NEW YORK, July 06, 2016 (GLOBE NEWSWIRE) -- Clinuvel Pharmaceuticals (ASX:CUV) (OTC:CLVLY) (XETRA:UR9) today announced a significant progression in its US regulatory pathway to gain market access for SCENESSE® (afamelanotide 16 mg) for the treatment of erythropoietic protoporphyria (EPP), a rare genetic disorder which forces patients to live in the dark due to absolute light intolerance. Clinuvel has concluded its US clinical program for EPP, evaluating the safety and efficacy of SCENESSE®.

The US FDA has granted SCENESSE® Fast Track designation1 following an initial review of data from Clinuvel’s clinical program. This designation recognises the severity of EPP and the unmet medical need in the US. Clinuvel commercially distributes SCENESSE® for the treatment of EPP in Europe.2

US FDA
The Fast Track designation enables Clinuvel to file scientific documents as part of a New Drug Application (NDA) on a rolling basis for US regulatory assessment. The dossier will also be eligible for priority review, an abbreviated review time of approximately six months. A pre-NDA meeting will take place shortly with the FDA to discuss the timing of the first filing of the complete scientific sections of the NDA dossier.

In 2008, the Office for Orphan Products of the FDA awarded SCENESSE® orphan designation for EPP. Significant advantages of the orphan designation include substantially lower NDA filing fees and market exclusivity for seven years following first market access in the US.

US EPP COMMUNITY
Since the commencement of a US clinical program for EPP in 2009 and the European marketing authorisation in 2014 Clinuvel has received a large number of requests from US EPP patients to access SCENESSE®. Clinuvel has openly communicated to patients and patient associations that it must await US regulatory clearance.

There is a strong network of expert EPP treatment centres in the US, coordinated by the US Porphyrias Consortium. These centres will now be notified of the expedited US regulatory process.

FDA SCIENTIFIC WORKSHOP ON EPP
FDA announced its intention to host a Scientific Workshop3 to learn more about EPP and drug treatment. The meeting is scheduled for 24 October 2016 in Silver Spring, Maryland.

COMMENTARY
“After 11 years of clinical evaluation of SCENESSE® and its European approval by the EMA for the treatment of EPP, we now have a clear runway ahead in the US,” Clinuvel’s Chair, Stan McLiesh said.

”The FDA has shown the vision to look at Clinuvel’s scientific data and now agree to a swift review process which will also involve patients and physicians in the evaluation process,” Mr McLiesh said.

“One can anticipate that the FDA will require Clinuvel to execute a Risk Evaluation and Mitigation Strategy (REMS) to ensure long term follow up of patient safety,” Clinuvel’s CEO, Dr Philippe Wolgen said. “A similar program is underway in Europe where SCENESSE® is commercially available under a strict post-marketing Risk Management Plan (RMP).

“We see increasing harmonisation between the European Medicines Agency and FDA relating to innovative pharmaceutical treatments. The Fast Track Designation now gives us the chance to prepare a rigid pharmacovigilance system for SCENESSE® in the US as is the case in Europe,” Dr Wolgen said.

1 Food and Drug Administration Modernization Act, 1997.

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