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Vertex Stock Up on Quick FDA OK For 3-Drug Cystic Fibrosis Therapy

Xconomy Boston — 

The FDA on Monday approved a Vertex Pharmaceuticals cystic fibrosis treatment that combines three drugs, a decision that comes three months after the company submitted its application and five months before the agency’s deadline to finish its review.

The Boston-based company saw its stock price rise 4 percent on the news to $185.

The Vertex (NASDAQ: VRTX) drug, Trikafta, combines two of its existing CF drugs, ivacaftor and tezacaftor, with elexacaftor, an experimental one—and was approved for about 90 percent of CF patients, many of whom had no approved therapeutic options, according to the FDA.

Cystic fibrosis, an inherited disorder, is caused by mutations to the gene that produces a protein that helps cells pump water in and out. In people with a mutation to the gene, organs, including the lungs and pancreas, often develop a thick layer of mucus that makes breathing difficult and can lead to bacterial infections. Trikafta was OK’d for those 12 years and older who have the most common CF mutation, called F508del.

Approval of Trikafta is the first for a three-drug combination treatment for CF, and it “puts even more distance between Vertex and any potential competitors,” SVB Leerink analyst Geoffrey Porges wrote in a research note.

Last year the company reported product revenue of $3 billion, up from $2.2 billion in 2017. Porges forecast Vertex’s new drug would become available in the first quarter of 2020, and reach $4.6 billion in sales by 2023 and $6.6 billion in 2025. Vertex set a $311,000 annual price for Trikafta—about 3 percent higher than SVB Leerink had estimated, he said.

Vertex’s third CF medicine, the two-drug combo ivacaftor/tezacaftor (Symdeko) approved last year, was listed at $292,000 per year.

Read more about the company’s cystic fibrosis franchise and its newly approved regimen here and here.