Vertex’ cystic fibrosis therapy Orkambi is in the spotlight again with the publication of two trials confirming the drug’s benefit in the New England Journal of Medicine.

Orkambi - a combination of lumacaftor and ivacaftor - is the first therapy that targets the underlying cause of CF, in about 45% of cases.

Data from the Phase III TRAFFIC and TRANSPORT studies show that the combination significantly improved lung function (as measured by FEV1) by 2.6%-4.0%, and reduced the rate of pulmonary exacerbations by 30%-39% in CF patients carrying two copies of the Phe508del CFTR mutation. 

Also, the therapy had “an acceptable side-effect profile”, with more than 93% of patients completing the assigned therapy regimen, the researchers noted.

Orkambi is widely being hailed as a breakthrough, and the findings have raised hopes that reducing the severity of the disease might ultimately translate into a survival benefit. “It is not a cure, but it is as remarkable and effective a drug as I have seen in my lifetime,” said Professor Stuart Elborn from the School of Medicine, Dentistry and Biomedical Sciences at Queen’s University Belfast, as reported by the Guardian.

The Cystic Fibrosis Foundation, which was involved in the Orkambi’s development, says the data is “a powerful validation that this drug is an important new CF treatment”.