Vertex’ new CF drugs may significantly increase US pharmacy benefit costs; report

Ground-breaking new cystic fibrosis treatments, that target the gene mutations causing the disease, will significantly increase health care costs in the USA, new research indicates.

This is the finding of new research by pharmacy benefit manager Prime Therapeutics. Cystic fibrosis is a genetic disease affecting multiple organ systems, most importantly the lungs and airways. An estimated 30,000 children and adults in the USA have the disease, which can lead to life-threatening lung infections and the inability to absorb nutrients from food. Some individuals may need a lung transplant because of progressive damage to the lungs.

In 2012, the first treatment to target a specific gene mutation, US biotech firm Vertex’s (Nasdaq: VTX) Kalydeco (ivacaftor) received US Food and Drug Administration approval. Ivacaftor treats a type of gene mutation found in about 4% of people with cystic fibrosis and costs $300,000 per year. A new combination drug – ivacaftor plus lumacaftor – is pending FDA approval for treatment of people aged 12 and older who have the gene mutation found in nearly 50% of people with cystic fibrosis. The price of the new drug is not yet public, but is widely expected to be similar to ivacaftor's price, ie, around $311,000 per-year.