CF Therapy Orkambi Approved in Europe

The European Commission has approved the cystic fibrosis drug Orkambi® for people with two copies of the F508del mutation ages 12 and older in the European Union.

Nov. 20, 2015 | 2 min read

In Europe, about 12,000 people with CF in this age group have two copies of F508del, the most common CF mutation.

Orkambi® targets the underlying cause of the disease -- a defective gene and its protein product, known as CFTR.

In July, the U.S. Food and Drug Administration approved Orkambi® for individuals with two copies of the F508del mutation ages 12 and older, representing about 8,500 people or nearly one-third of those with CF in the United States.

The drug is the second to treat the faulty CFTR protein in CF. The first, ivacaftor (Kalydeco®), has been approved for people with certain rare mutations of CF.

Orkambi® is a combination of ivacaftor and lumacaftor. In people with two copies of F508del, the drug improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease.

Orkambi® was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific and funding support from the CF Foundation.