Vertex Wins EU Approval for Cystic Fibrosis Drug

Xconomy Boston — 

[Updated: 2:30 pm ET] Vertex Pharmaceuticals made big news in the U.S. cystic fibrosis community six months ago when it won FDA approval for a groundbreaking drug, and now it’s taking the product to Europe.

The Cambridge, MA-based biotech company (NASDAQ: VRTX), which has significant operations in San Diego, said today it has gotten clearance from the European Commission to start selling ivacaftor (Kalydeco) as a new treatment for patients ages 6 and older who have a gene mutation called G551D. This is a rare form of cystic fibrosis, which affects about 1,200 patients in the U.S., and another 1,100 in Europe, Vertex said. The regulatory approval marks the first time Vertex has been cleared to market a product in Europe.

The new product, developed through a collaboration between Vertex and the Cystic Fibrosis Foundation, has been a watershed for the field. The disease comes from mutations to a gene called CFTR, which causes poor transfer of water and salt across cell membranes, and which leads to the buildup of thick, sticky mucus in the lungs that ends up suffocating people once they reach middle age. Doctors have long sought to treat the symptoms, but Vertex’s drug is the first therapy shown to work by altering an underlying disease-related protein.

About half of the U.S. population eligible for the new drug started taking it in its first quarter on the market, Vertex has said. The patients flocked to the product based on results the company showed in its pivotal clinical trial. The key 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. Researchers also saw significant improvements in reducing cough, sputum production, weight gain, and the reduction of worrisome lung exacerbations. Side effects included headache, upper respiratory tract infections, nasal congestion, rash, and dizziness, Vertex said.

Although the Vertex drug is only approved for a small segment of the total 30,000 cystic fibrosis patients in the U.S. and 35,000 in Europe, it still has potential to be a big seller because of its high price. The drug is priced at $294,000 per patient per year in the U.S., and it is intended to be taken by each patient for many years. Vertex didn’t say in today’s statement what the price will be in various European countries.

[Updated company comment] Vertex spokeswoman Dawn Kalmar said in an email that the company is working to make Kalydeco available “as quickly as possible” across Europe, but the process for getting reimbursement differs from country to country. “For example, Germany allows new products to be reimbursed upon approval,” Kalmar said. “The U.K. and Ireland allow sales, but broad use is not expected to be achieved until pricing is negotiated. Broad access and reimbursement in France can take 6 to 9 months post EMA approval. Pricing is set on a country-by-country basis and we are in discussions with health authorities about price.”

The company could achieve peak U.S. sales of about $550 million in the G551D population, according to analyst Mark Schoenebaum of ISI Group. Many analysts are eagerly awaiting further clinical trial results to see if Vertex can extend the benefits of the product to a broader swath of cystic fibrosis patients, by giving it in combination with another experimental drug called VX-809.