Clinuvel scores long-awaited FDA win for rare skin disease implant Scenesse

fda
The FDA approved Clinuvel's Scenesse to treat a rare skin disorder. (FDA)

It wasn’t so long ago that Australian drugmaker Clinuvel spurned a buyout attempt by Retrophin and its wayward then-CEO Martin Shkreli, preferring to soldier on alone with its sole drug candidate. Now, that drug has scored its FDA approval, and Clinuvel's poised for a big leap in value.

Wednesday, the FDA gave its blessing to Scenesse, an under-the-skin implant to treat erythropoietic protoporphyria (EPP), a disease that causes itching, burning and scarring of the skin on contact with sunlight.

Cleared in Europe in 2014, Scenesse is now the first FDA-approved treatment for EPP, which affects between 75,000 and 200,000 individuals worldwide, Clinuvel said. The company is studying the implant for other uses, including the pigmentation disorder vitiligo, and has three other preclinical programs working.

On-Demand Webinar

De-Risking the Solid Form Landscape of an API

This presentation will discuss how predictable stability and solubility can minimize development timelines and cost. Attend to hear about two case studies exemplifying the importance of understanding the hydration space of an API and how hydrate formation may be avoided by development of a robust crystallization procedure.

In a statement (PDF), Clinuvel CEO Philippe Wolgen said the drugmaker intends to push ahead with U.S. and EU regulators to broaden the market for Scenesse, its only approved treatment. Wolgen told Bloomberg the company plans to sell Scenesse directly to U.S. hospitals and clinics—where the implants will be placed—at a price point that matches that in other countries, a move he called “quite alien to our industry.”

RELATED: Retrophin Proposes Acquisition of Clinuvel Pharmaceuticals

On the heels of Scenesse’s U.S. approval, Clinuvel stock was trading up 57% in Sydney—its largest jump since 2014—at a price of $44.39 Australian ($29.94 U.S.).

Clinuvel’s road to U.S. approval was a lengthy one after the FDA initially granted orphan drug status to Scenesse back in 2008.

In 2014, deal-obsessed Shkreli and Retrophin pitched Clinuvel on a full takeover at a 17.5% premium, hoping to shepherd the long-gestating rare disease treatment to approval. Instead of taking the offer, Clinuvel continued on alone and scored its EU nod the same year.

Without the deal, Retrophin went south. The biotech’s investors sued Shkreli in 2015 for allegedly taking Retrophin public to give stock to investors from his then-defunct hedge fund MSMB. The biotech later agreed to pay out $3 million in an investor lawsuit alleging the company did not disclose transactions that benefited Shkreli, who was later convicted of securities fraud and conspiracy in April 2018 and sentenced to seven years in prison.

Meanwhile, following its EU approval in 2014, Clinuvel waited two years before the FDA asked for a full set of Scenesse’s clinical trials, leading to priority review status in January of this year.

Despite the win Wednesday, Wolgen said hard work is still ahead for Clinuvel as it moves on to other approvals for its forerunner med.

“I am confident that our teams will understand going forward that art and skills will be required to execute against due dates while containing the costs,” Wolgen said in a statement. “If the past had been arduous, the hard labour is just about to start.”

Suggested Articles

J&J's talc woes deepened Friday after the FDA turned up "sub-trace" levels of asbestos one bottle of the company's baby powder,…

Another major drugmaker is recalling in the U.K. 10 batches of its Zantac generics because they contain a possible carcinogen.

With diagnosis rates on the rise, Pfizer's Vyndaqel franchise could collect $157 million in 2019 U.S. sales, well above consensus, SVB Leerink says.