Amicus Therapeutics to acquire Scioderm in deal potentially worth about $950 million

Amicus Therapeutics signed a definitive agreement to buy the biopharmaceutical company Scioderm, boosting its rare-disease portfolio with the latter's experimental topical cream Zorblisa, which is currently in Phase III testing for epidermolysis bullosa, the companies announced Monday. Under the deal, Amicus will pay Scioderm $229 million in a mix of cash and stock, with Scioderm also eligible to receive an additional $361 million if clinical and regulatory milestones are reached, as well as $257 million upon the achievement of certain sales targets. Further, if Zorblisa is approved, Amicus said a priority review voucher will be requested, adding that it will pay Scioderm up to a maximum of $100 million from the proceeds of an eventual possible sale of the voucher.

The acquisition, which is expected to be completed in the third quarter, "is a major step forward toward our strategic vision and is transformative for the epidermolysis bullosa…community, as well as the shareholders of Amicus and Scioderm," remarked Amicus CEO John Crowley, who also serves on Scioderm's board. Crowley said Amicus is "well-positioned to rapidly complete the clinical development of Zorblisa and to make [the treatment] commercially available for all epidermolysis bullosa patients as quickly as possible." He also noted that "when combined with migalastat for Fabry disease and ATB200 for Pompe disease, this acquisition solidly positions Amicus as a leading global rare-disease company."

Amicus said the Phase III SD-005 study of Zorblisa is currently enrolling paediatric and adult patients with epidermolysis bullosa in support of global regulatory submissions, with data anticipated in the first half of 2016. The company said the FDA agreed to a rolling submission for the therapy beginning in the fourth quarter, while EU regulators have accepted Scioderm's paediatric investigation plan. Zorblisa, formerly known as SD-101, was awarded breakthrough therapy status by the FDA for the treatment of inherited epidermolysis bullosa in 2013 based on positive Phase II proof-of-concept data.

According to Amicus, Zorblisa represents a possible first-to-market therapy with the potential to reach $1 billion or more in global sales. The company said the current standard of care for epidermolysis bullosa consists of palliative treatments costing between $10 000 and $15 000 per month. Amicus estimated that 30 000 to over 40 000 people are affected by the disease in major markets.

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