Australian company surges on FDA approval

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The share price of Australian company Clinuvel Pharmaceuticals (ASX:CUV) surged over 60 per cent yesterday after the company confirmed FDA approval of its SCENESSE (afamelanotide 16mg) for the treatment of erythropoietic protoporphyria (EPP).

EPP is a genetic disorder that causes absolute light intolerance and forces patients to lead an indoor or nocturnal existence.

SCENESSE is the first global systemic photoprotective drug for the treatment of patients with EPP. It acts as a potent anti-oxidative and melanogenic agent providing total surface photoprotection. It was approved in Europe in 2014.

Investors cheered the news with the company's market capitalisation surging to $2.2 billion.

SCENESSE had a long path to approval in the US, starting with clinical trials in Europe in 2006, followed by the FDA granting Orphan Drug Designation in 2008.

The company commenced a phase 2 clinical trial (CUV030) in 2010 followed by a phase 3 trial (CUV039) that was completed in 2013.

The FDA awarded SCENESSE fast-track designation in 2017 and priority review in early 2019.

“I cannot start to describe what it means to dedicate a large portion of one’s professional life to a single molecule and for one group of patients, while not knowing the regulatory outcome,” said CLINUVEL’s chief scientific officer, Dr Dennis Wright.

“However, we kept our awareness of the regulatory risks with a first-in-class melanocortin agonist as we faced the subsequent regulatory challenges. The team kept going.

“The outcome today is greatest for the patients and their families who kept asking us to continue the R&D of SCENESSE despite the obstacles we faced. The approval of SCENESSE today is deserved and based on its safety and medical benefits to patients,” added Dr Wright.

“This is one day on which all interests converge and history is written both for the US EPP patient community and investors who have actively supported our mission for the last 14 years,” said CEO Dr Philippe Wolgen.

“The FDA approval of SCENESSE as a new molecular entity and medical innovation is memorable for this Company and for the Australian life science sector.

“At this time, it is most appropriate to express my gratitude to the Board of Directors and CLINUVEL staff who never hesitated or lost the belief in a strategy that was devised in 2005 and which was religiously pursued by all. Despite numerous setbacks, resistance and delays, the commitment of my staff is quite unique, and I publicly wish to thank them all.

"In curtailing costs and maximising data required, we stressed throughout the safety aspects of the novel pharmaceutical technology. In working together with select scientific and clinical experts we gained understanding and feedback on our chosen course."