Combo Cystic Fibrosis Therapy Approved

The US Food and Drug Administration (FDA) on Thursday approved Orkambi (lumacaftor/ivacaftor/Vertex) to treat cystic fibrosis patients age 12 and older with two copies of the F508del mutation in the CFTR gene.

Orkambi was cleared to be taken as one pill, since it combined the active ingredient in Kalydeco, ivacaftor, with lumacaftor.

Researchers tested the safety and efficacy of Orkambi in two phase 3 studies involving 1,108 participants, which demonstrated a nearly three percentage point improvement in lung function compared to placebo.

The most common adverse events reported included shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rashes.

The team underscored the safety and efficacy of Orkambi was only tested in cystic fibrosis patients with the F508del mutation, not in those with other mutations or younger than 12 years old.

Deciding on cost, Vertex will potentially set the gross price prior to insurance discounts in the $230,000 - $250,000 range annually.

Orkambi will soon be available to approximately 8,500 US patients — more than 2,000 who already take Kalydeco, which was reported to target a different genetic mutation triggering cystic fibrosis.

John Jenkins, MD, director of the Office of New Drugs, Center for Drug Evaluation and Research, said in a press release, “Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis.”