ADVERTISEMENT

Vertex Cystic Fibrosis Drug Gets FDA Approval Ahead of Schedule

Vertex Cystic Fibrosis Drug Gets FDA Approval Ahead of Schedule

(Bloomberg) -- Vertex Pharmaceuticals Inc. won approval from the U.S. Food and Drug Administration for a new three-drug combination that could help 90% of patients with cystic fibrosis, a development that could extend treatment options to those with no approved therapy.

The approval -- five months ahead of schedule -- sent the shares of Vertex surging as much as 4.9%. Analysts expect the drug to have more than $3 billion in annual sales in 2023, according an average of six estimates compiled by Bloomberg.

The new treatment, called Trikafta, combines two drugs that make up one of Vertex’s existing medicines, Symdeko, with a third one that will allow it to treat an additional set of patients. Vertex estimates that about 6,000 additional cystic fibrosis patients in the U.S. may be eligible for treatment with the new drug.

The new combination therapy will cost $311,503 a year, or roughly $24,000 for four weeks, the company said in a securities filing.

“In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options,” FDA Acting Commissioner Ned Sharpless said in a statement announcing the approval. The agency used “all available programs” to expedite the review and approval as efficiently as possible.

Cystic fibrosis is a life-threatening genetic disease that causes mucous to build up in the lungs and other parts of the body. It severely reduces life expectancy.

“This is what we have been building for for the last two decades,” said Michael Boyle, senior vice president for therapeutics development at the Cystic Fibrosis Foundation. “As we start to have more and more people on these efficient treatments, there is no doubt the life expectancy will continue to increase.”

The foundation sponsored early research that led to the new class of drugs, called modulators, that directly attack the disease’s cause.

To contact the reporter on this story: Robert Langreth in New York at rlangreth@bloomberg.net

To contact the editors responsible for this story: Drew Armstrong at darmstrong17@bloomberg.net, Mark Schoifet, Timothy Annett

©2019 Bloomberg L.P.