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Orkambi FDA Approval History

Last updated by Judith Stewart, BPharm on Jan 10, 2021.

FDA Approved: Yes (First approved July 2, 2015)
Brand name: Orkambi
Generic name: ivacaftor and lumacaftor
Dosage form: Tablets and Oral Granules
Company: Vertex Pharmaceuticals Incorporated
Treatment for: Cystic Fibrosis

Orkambi (ivacaftor and lumacaftor) is a CFTR potentiator and CFTR corrector combination for the treatment of the underlying cause of cystic fibrosis in patients 2 years and older with two copies of the F508del mutation in their CFTR gene.

Cystic fibrosis (CF) is a rare genetic disease that leads to a deadly buildup of thick mucus in the lungs and other organs. It is caused by a defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) protein resulting from mutations in the CFTR gene. Orkambi is approved for use in patients 2 years and older who have two copies of the F508del mutation in the CFTR gene, accounting for about half of all CF cases.

Orkambi contains ivacaftor (Kalydeco), FDA approved to treat a smaller subset of CF patients with different gene mutations, and lumacaftor.

The safety and efficacy of Orkambi was studied in two double-blind, placebo-controlled clinical trials. In both studies, the participants who took Orkambi demonstrated improved lung function, reductions in pulmonary exacerbations and improvements in body mass index (BMI) compared to those who took the placebo.

Orkambi is available as oral tablets for use in adults and children over 6 years, and oral granules for use in children between 2 and 5 years of age. Doses are taken twice daily (every 12 hours) with fat-containing food.

Common side effects include shortness of breath, upper respiratory tract infection (common cold) and gastrointestinal symptoms (including nausea, diarrhea, or gas). Menstrual abnormalities such as increased bleeding have been reported among women of childbearing age.

Development Timeline for Orkambi

Aug  7, 2018Approval  FDA Approves Expanded Indication for Orkambi (lumacaftor/ivacaftor) in Children Ages 2-5 Years
Sep 28, 2016Approval  FDA Approves Orkambi (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation
Jul  2, 2015Approval  FDA Approves Orkambi (lumacaftor/ivacaftor) for Cystic Fibrosis
May 12, 2015FDA Advisory Panel Voted 12 to 1 to Recommend Approval of Orkambi (lumacaftor/ivacaftor) for Cystic Fibrosis F508del Mutation
Nov  5, 2014Vertex Submits NDA for Lumacaftor in Combination with Ivacaftor for Cystic Fibrosis F508del Mutation

Further information

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