Vertex Seeks FDA Green Light for Cystic Fibrosis Drug, Second Potential Hit of Big Year

Xconomy Boston — 

Cambridge, MA-based Vertex Pharmaceuticals is looking to be more than another one-hit biotech wonder. The company, best known for its drug for hepatitis C introduced earlier this year, is now vying for FDA approval of a second treatment that it hopes will shake up the way physicians treat cystic fibrosis, a deadly genetic disease.

Vertex (NASDAQ: VRTX) said today it has turned in its new drug application to the FDA for clearance to sell ivacaftor (Kalydeco, aka VX-770) as a new treatment for a rare form of cystic fibrosis in the U.S. The drug, pronounced kuh-LYE-deh-koh, is designed to treat about 4 percent of the 30,000 or so people in the U.S. with cystic fibrosis who have a mutation of a gene known as G551D. The company is seeking a six-month expedited regulatory review, instead of the usual 10-month review. The FDA sometimes provides faster reviews for groundbreaking new medicines.

The new cystic fibrosis drug has been highly anticipated for years, and the application to the FDA is based heavily on clinical trial results that arrived in February. The study of 161 patients found that patients on the Vertex drug had about a 10 percent absolute improvement in their ability to force out air from their lungs in one second—a common measure of success in treating lung disease-compared with those on a placebo. The effect lasted over the entire 48-week course of the study. Side effects included headache, upper respiratory tract infections, nasal congestion, rash, and dizziness, although more patients on the placebo group dropped out of the study early because of side effects, Vertex said.

While the 10 percent absolute improvement in breathing ability for people with a deadly lung disease might not sound like much, the result is a big step forward for the disease. Cystic fibrosis, the result of a genetic mutation, causes the poor transfer of water and salt across cell membranes, which leads to the buildup of thick, sticky mucus in the lungs. That effectively suffocates people over time, and often ends up killing people in their late 30s or early 40s. Doctors currently treat the symptoms of the disease, but Vertex’s drug could become the first FDA-approved therapy that works by altering an underlying disease-related protein.

The drug was developed as part of a 13-year long collaboration with the Cystic Fibrosis Foundation, which put more than $70 million into the development program, along with Vertex, and the drug’s original developer that was acquired by Vertex—San Diego-based Aurora Biosciences.

The new CF drug will surely be expensive, given its impact for a small group of patients with a deadly disease. Vertex, like all drug companies, isn’t saying what the price will be until approval. But, naturally, there’s a fair amount of speculation about what the market will pay for such a drug. Thomas Russo, an analyst with Robert W. Baird, quoted a physician in a June research report who said the clinical results for the drug were the best she has seen in 23 years of treating CF. Russo estimates the drug will cost about $150,000 a year, and will generate about $700 million a year in annual revenues over time.

“We are quite comfortable that our model is very conservative and that VRTX’s CF franchise is still a story about potentially very sizable upside,” Russo wrote in the June 16 note.

Vertex said it plans to turn in its application for European Union approval of the new CF compound by the end of this month.

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