Amicus Therapeutics said today it will acquire Scioderm for up to $847 million in cash and stock, in a deal that expands the buyer’s pipeline of drugs for rare and orphan diseases with a Phase III candidate set to begin a rolling NDA later this year.

Scioderm’s lead product Zorblisa™ is a potential first-to-market therapy for epidermolysis bullosa (EB), and in Phase III development for treatment of the skin effects associated with the rare disease.

Scioderm is enrolling pediatric and adult EB patients across all major subtypes for the Phase III SD-005 study, with the goal of generating data to support global regulatory approvals. The study’s primary outcome measure is complete target wound closure within two months. Estimated enrollment in the study is 130 patients, with an estimated study completion date of January 2016, according to the study’s page on, last updated August 25.

The FDA has agreed to a rolling NDA submission for Zorblisa beginning in the fourth quarter, while European regulators have approved a pediatric investigation plan for the drug candidate. Data from SD-005 is anticipated in the first half of 2016. Zorblisa received one of FDA’s first-ever Breakthrough Therapy designations in 2013, based on positive Phase II proof-of-concept data for the treatment of lesions in patients suffering with EB.

“Both Amicus and Scioderm are wholly and passionately focused on patients with rare diseases, and share a common vision and similar values,” Robert Ryan, Ph.D., Scioderm’s president and CEO, said in a statement. Dr. Ryan will join Amicus’ senior leadership team.

Amicus has estimated that Zorblisa may represent a potential “$1 billion-plus global market opportunity,” since the drug represents an advance over the current standard of care of palliative treatments, which according to the company cost $10,000 to $15,000 per month, and mainly consist of bandaging, treating the open wounds to prevent infection, and trying to manage patients' pain.

An estimated 30,000 to 40,000+ people are currently diagnosed with EB in major markets, Amicus adds.

“This acquisition is a major step forward toward our strategic vision and is transformative for the Epidermolysis Bullosa, or EB, community as well as the shareholders of Amicus and Scioderm,” Amicus chairman and CEO John F. Crowley, who is also a board member of Scioderm. “We believe we are well-positioned to rapidly complete the clinical development of Zorblisa and to make Zorblisa commercially available for all EB patients as quickly as possible.”

Zorblisa joins Amicus’ pipeline of rare disease drugs, which is led by Galafold (migalastat, formerly AT1001). The small molecule treatment is being developed as an oral monotherapy for Fabry disease patients with α-Gal A mutations who are amenable to the pharmacological chaperone—and in combination with enzyme replacement therapy for all other patients through Amicus’ Chaperone-Advanced Replacement Therapy (CHART™) platform.

The European Medicines Agency is reviewing Amicus’ Marketing Authorization Application (MAA) for Galafold, while the company has said it plans to submit a U.S. marketing application for the drug in the second half of this year.

Amicus’ pipeline also includes ATB200 for Pompe disease, an enzyme replacement therapy also developed through the CHART platform and set to advance into the clinic by year’s end, with potential for a Phase III study next year.

“When combined with migalastat for Fabry disease and ATB200 for Pompe disease, this acquisition solidly positions Amicus as a leading global rare disease company dedicated to bringing substantial value to patients and shareholders,” Crowley added.

Amicus said it agreed to pay Scioderm shareholders $229 million—of which $125 million will be paid in cash and $104 million will be paid through the issuance of 7 million newly issued Amicus shares. Amicus plans to finance the acquisition through cash on hand, and has a $50 million debt commitment from Redmile Group.

Amicus also agreed to pay up to an additional $361 million to Scioderm shareholders in cash or stock upon achievement of clinical and regulatory milestones, and $257 million to Scioderm shareholders in cash or stock upon achievement of certain sales milestones.

If Zorblisa is approved, EB qualifies as a rare pediatric disease and a Priority Review Voucher will be requested. If the Priority Review voucher is obtained and subsequently sold, Amicus will pay Scioderm shareholders $100 million or 50% of the proceeds of such sale, whichever is less.

The deal is subject to customary conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. The boards of both Amicus and Scioderm have approved the transaction, with the companies saying they anticipate the transaction will be completed by the end of the third quarter.

Privately held Scioderm was financed initially in 2013 by Morgenthaler Ventures and Technology Partners, followed by a subsequent financing led by Redmile Group and included the initial investors.

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