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Vertex cystic fibrosis combo wins backing of U.S. advisers

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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.

Lung function

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.

See also: Cost to develop a drug more than doubles

The FDA didn’t require Vertex to study whether Kalydeco alone might work after a trial used for earlier approval showed it might not be effective for the most common form of the disease. That trial was meant to measure safety, not whether the drug works.

Prescribing information on Kalydeco advises that it isn’t effective for the most common form of cystic fibrosis.

“That probably was a bit of a hasty decision being made on a clinical trial that wasn’t designed for efficacy,” Anthony Durmowicz, a clinical team leader at the FDA, said during the meeting.

Expanded use

The FDA approved Kalydeco in 2012 for use in about 1,200 U.S. cystic fibrosis patients who have a certain genetic mutation. Since then, the drug’s use has been expanded to about 2,000 people. By combining Kalydeco with lumacaftor, Vertex is seeking to treat an additional 8,500 patients who are 12 and older and have the most common form of the disease.

Cystic fibrosis affects 30,000 children and adults in the U.S., according to the Cystic Fibrosis Foundation. It can be a fatal disease that causes patients’ lungs and other organs to clog with thick, sticky mucus.

Orkambi is targeted at patients with two copies of the F508del mutation in the CFTR gene, defects in which lead to cystic fibrosis. About 50 percent of people with the condition in the U.S. have two copies of the F508del mutation, according to the Cystic Fibrosis Foundation.

Kalydeco costs about $300,000 per patient per year, and some analysts are saying Orkambi could cost more than $200,000 per patient per year.

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