Vertex Pharmaceuticals, which is based out of Boston, has announced that a combination of two drugs made some improvements to the lung capacity of patients with cystic fibrosis. The drug combination—two doses of lumacaftor added to Vertex’s existing CF drug Kalydeco—prevented infections that would’ve landed patients in the hospital and required antibiotics. The combination also helped patients gain weight.
The drug combination is effective, but it is costly—the therapy is estimated to cost as much as $160,000 per patient for a year’s worth of treatment. Still, that number is a significant drop off from the current cost of Kalydeco, which currently costs $307,000 per patient per year in the US. The Cystic Fibrosis Foundation funded early Kalydeco research and gets a royalty on all Vertex drugs. Vertex maintains that the Foundation plays no part in the pricing of Vertex drugs.
Bonnie Ramsey, the professor of pediatrics at the University of Washington, was one of the investigational leaders of a Vertex trial on the drug combination. “I was actually very pleasantly surprised,” said Ramsey. “I was very pleased, and I’m usually very critical.”
Experts believe that the news will increase the price of Vertex’s shares. The next step for Vertex is getting the Kalydeco and lumacaftor drug combination approved by the FDA. The company has already stated that it hopes to file for approval by the fourth quarter of 2014, with final approval of the treatment combination possibly coming in the subsequent eight months.
Kalydeco was approved for treating patients with CF by the FDA in 2012.