Clinuvel completes Scenesse FDA filing

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Australian company Clinuvel has announced that the US FDA has begun a review of its therapy 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.

Clinuvel has spent the past 14 years developing the therapy - SCENESSE or afamelanotide 16mg.

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,. 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 approved in Europe for adult EPP patients, uses the body’s own defensive systems to protect EPP patients from light. 

The therapy, administered as a subcutaneous dose every two months, activates melanin (pigment) in skin.  This acts as a physical protective barrier to shield the chemicals in the blood from light.

Clinuvel said it expects the FDA to complete the review in 2019.

“The innovation developed as SCENESSE activates an umbrella of melanin which shields patients’ skin cells from light sources,” said chief scientific officer Dr Dennis Wright.

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,” said Professor Minder. “Therefore, I am delighted that this treatment finally nears the point of becoming available to all American EPP patients.”

The company said 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," it said. "Two Phase III trials, from the US and Europe, were published in the New England Journal of Medicine, noting good tolerance to the drug."

“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,” said Dr Wright. “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.”