Bloomberg) — Vertex Pharmaceuticals Inc.’s experimental cystic fibrosis combination should be approved by regulators at the U.S. Food and Drug Administration (FDA), a panel of outside advisers said.
The panel voted 12-1 that a combination of an experimental drug, lumacaftor, and Vertex’s Kalydeco, which is already available, should be cleared for patients with the most common form of the lung disease. Vertex proposes calling the combination Orkambi.
FDA staff last week questioned whether the combination works any better than Kalydeco alone. Members of the panel voted that because of the design of the trial, they couldn’t determine whether lumacaftor added benefit to Orkambi. The agency is scheduled to decide by July 5 whether to approve the combination. Analysts estimate sales of Orkambi could reach $3.41 billion in 2018.
“Given the FDA agreed to this design, it wouldn’t be fair to the patient to deprive them of this now,” Erica Brittain, a panel member and statistician at the National Institute of Allergy and Infectious Diseases, said during the meeting. “Hopefully they can still possibly do this study post-approval. I also hope this will be a lesson learned for combination products.”
Vertex shares gained 7 percent to $132.75 after the market closed in New York. Trading in the stock was halted all day during the meeting.
While the panel struggled with whether lumacaftor added benefit to Orkambi, it determined that the therapy improves lung function, so the individual contributions of the drugs may be less relevant. The FDA raised the question after seeing similar effects on lung function between the combination and an earlier trial on Kalydeco. Six members of the panel voted that they couldn’t determine whether lumacaftor added to the benefit of Orkambi. Four voted that the data didn’t show lumacaftor added benefit, and three voted that it did.
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