Vertex wins once-rejected Kalydeco label expansion, bumping guidance by $30M-plus

The FDA has approved a label expansion for Vertex's Kalydeco that affects more than 900 U.S. cystic fibrosis patients.

Last February, the FDA nixed Vertex’s bid to expand cystic fibrosis med Kalydeco to patients with specific mutations in the CFTR gene. On Wednesday, though, the Cambridge drugmaker finally got what it wanted.

Regulators approved the orphan drug to treat patients age 2 and older who have one of 23 residual function CFTR mutations—a population that numbers more than 900 in the U.S., Vertex said. 

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And for a company selling an expensive med into small patient pools, even a comparatively tiny influx of patients means a revenue boost. Vertex upped its 2017 Kalydeco guidance to between $740 million and $770 million, versus the $710 million to $730 million range it previously predicted.

Whatever Kalydeco sales haul the new approval posts this year, though, will likely shrink next year, Leerink analyst Geoffrey Porges wrote in a note to clients. That’s because Vertex is on track to debut a combo of Kalydeco and candidate med tezacaftor for the residual function group; in late March, the drugmaker unveiled that two phase 3 studies had nailed their primary endpoints.

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“In one sense this approval pulls forward the revenue ramp in a portion of the residual function population that would otherwise have accumulated to treatment after the tez/iva approval,” Porges said.

Meanwhile, it shouldn’t take long for Vertex to get the word out on the new Kalydeco go-ahead. Porges expects Vertex to “immediately” present the rubber stamp to payers, physicians and patients, and “reimbursement should be relatively rapid given the small patient population involved,” he said. He expects 65% to 70% of eligible patients to be on Kalydeco by 2017’s close, he added.