Here's what a buyout deal for a RARE disease is wo

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Amicus Therapeutics has stepped in to snap up the late-stage rare disease biotech Scioderm, beefing up its orphan drug pipeline in exchange for $229 million in stock and cash along with a promise of up to $618 million more for meeting a slate of milestones.

The buyout leaves Amicus ($FOLD) with its lead drug Galafold under regulatory review, a new drug that could be filed in the near term and a third program entering the clinic--with CEO John Crowley prepping for more deals as the company builds out a global commercial team.

Amicus completes this buyout a little more than a month after Scioderm started a rolling submission of its NDA for Zorblisa (SD-101), an experimental drug for epidermolysis bullosa, a condition that leaves children's skin papery thin and fragile, subject to tearing and blistering. Most patients--and there are some 30,000 to 40,000 in the world's major drug markets--die before the age of 30.

Data from the late-stage study is due in the first half of next year, with Amicus looking for a regulatory green light on a drug Crowley believes could be a blockbuster.

The market for Zorblisa, says the CEO, is worth "a billion dollars-plus." Adds Crowley: "We want to be one of the leading biotech companies focused on rare diseases."

Not that the deal is without risk. TheStreet notes that Zorblisa failed its Phase IIb study, with a small subgroup of patients in the top dose achieving statistical significance compared to a placebo. That top dose is being taken into the Phase III with the blessing of regulators.

There's an added bonus involved in this deal. If Zorblisa is approved, the owners could qualify for a priority review voucher--an asset that's been worth hundreds of millions of dollars in recent deals. Amicus will pay Scioderm's investors either $100 million or half the sales price for the voucher, whichever is less.

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