Biotechnology, Business, Drug Development

Stalled at the FDA, Amicus adds a preclinical rare disease drug in back-ended $90M deal

Amicus Therapeutics $FOLD has snagged a preclinical drug in a small biotech buyout, beefing up its slim pipeline at a time that the company is still assessing just how badly its lead drug may have stalled in front of the FDA.

The Cranbury, NJ-based biotech acquired MiaMed for $1.8 million in cash, $4.7 million in Amicus stock and up to $83 million in development and commercialization milestones. The bulk of the money is back-ended to the commercialization stage.

For that Amicus gets a preclinical program aimed at restoring missing CDLK5 protein, an extremely rare condition that interrupts normal brain development, with catastrophic results.

Amicus won a European approval for Galafold (migalastat) in May, which partly helped make up for an embarrassing reversal last year when its sunny outlook on an FDA approval suddenly clouded after it did a retake on the agency’s guidance. Amicus says it should be close now in determining exactly what it will need to do to get an FDA OK, and whether that includes conducting a new trial.

Amicus shares never have recovered from the drubbing that followed the setback at the FDA. But the company hasn’t gone comatose, either.

“Most children with CDKL5 have frequent seizures that begin shortly after birth,” said CEO John Crowley. “They experience severe impairment in neurological development, and many of them are unable to walk, talk or care for themselves.”


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RAPS Regulatory Convergence 2017