FDA to Review Australian Drug for Genetic Disorder of Absolute Light Intolerance

6/25/2018, 12:00 PM (Source: GlobeNewswire)

MELBOURNE, Australia, June 25, 2018 (GLOBE NEWSWIRE) -- In a landmark case, the US Food and Drug Administration (FDA) has begun a review of an innovative drug developed for patients who carry a rare genetic blood disorder known as erythropoietic protoporphyria (EPP). EPP patients suffer extreme biochemical reactions when they expose their skin to any light source, particularly the spectrum of visible light, restricting their ability to function properly and forcing them to lead an isolated life in the dark.

The pharmaceutical innovation – known as SCENESSE® or afamelanotide 16mg – was developed for EPP over the past 14 years by the Australian pharmaceutical company CLINUVEL.

Due to a genetic defect, EPP patients carry a deficiency in one of their key enzymes to produce heme, and consequently accumulate a chemical substance in their blood which immediately reacts when patients are exposed to light, including sun, ambient and artificial sources. Patients incur physical burns and ulcers, and are in a state of crisis following light exposure, summarized as phototoxicity. This usually occurs within minutes of exposure to bright lights, especially sunlight. Phototoxic reactions last from days to weeks, and are unresponsive to any other medications.

SCENESSE®, which is already authorized in Europe for adult EPP patients, uses the body’s own defensive systems to protect EPP patients from light.1 The drug, administered as a subcutaneous dose every two months, activates melanin (pigment) in skin, which acts as a physical protective barrier to shield the chemicals in the blood from light. CLINUVEL has now submitted a new drug application to the FDA requesting SCENESSE® be authorized for distribution in the US, and expects the review to be completed in 2019.

The innovation developed as SCENESSE® activates an umbrella of melanin which shields patients’ skin cells from light sources,” CLINUVEL’s Chief Scientific Officer Dr Dennis Wright said.

Professor Elisabeth Minder, head of Biochemical Laboratory Analytics at Triemli Hospital in Zurich, was involved in the first clinical trials of the product in EPP and continues to treat EPP patients.

My patients tell me, since 2006, that this treatment facilitates a life which was unthinkable for them and their immediate families,” Prof Minder said. “Therefore, I am delighted that this treatment finally nears the point of becoming available to all American EPP patients.”

Over 800 patients have received SCENESSE®, including 350 EPP patients in clinical trials. Results from these trials showed the drug is able to reduce the number and severity of phototoxic reactions, and allows a life which patients could never lead before. Two Phase III trials, from the US and Europe, were published in the New England Journal of Medicine, noting good tolerance to the drug.2 CLINUVEL continues to monitor the drug’s ongoing use in Europe, and will establish a similar program in the US, if approved.

We are proud to have worked so hard to be in a position where SCENESSE® may become available to US EPP patients as early as next year,” Dr Wright said. “EPP is a significantly neglected disease, one which our team have focused on since 2005. It is fantastic news for US patients and their families that there is finally hope of receiving a treatment that will allow them to come out of the dark. The US EPP patients have proven to be very motivated during the clinical trials and deserve adequate medical care.”

1 SCENESSE® (afamelanotide 16mg) is approved in Europe as an orphan medicinal product for the prevention of phototoxicity in adult patients with EPP. Information on the product can be found on CLINUVEL’s website at www.clinuvel.com.
2 Langendonk et al (2015). Afamelanotide for Erythropoietic Protoporphyria. NEJM. 373:48-59.

Media inquiries
Europe:               Lachlan Hay, CLINUVEL (UK) LTD. +44 1372 860 765                                Lachlan.Hay@clinuvel.com
USA:                   Terri Clevenger, Continuum Health Communications, +1 (203) 227-0209,   tclevenger@continuumhealthcom.com
Australia:            Rudi Michelson, Monsoon Communications, +61 3 9620 3333,                    rudim@monsoon.com.au

CLINUVEL PHARMACEUTICALS LTD (ASX:CUV) (NASDAQ INTERNATIONAL DESIGNATION ADR:CLVLY) (XETRA-DAX:UR9) is a global biopharmaceutical company focused on developing and delivering treatments for patients with a range of severe genetic and skin disorders. As pioneers in photomedicine and understanding the interaction of light and human biology, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for photoprotection and repigmentation. These patient groups range in size from 5,000 to 45 million worldwide. 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). More information on EPP can be found at http://www.epp.care.

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 to the Australian Securities Exchange and to press 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.

Level 6, 15 Queen Street              T +61 3 9660 4900         www.clinuvel.com
Melbourne, Victoria 3000              F +61 3 9660 4999

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