Orkambi Approval History
- FDA approved: Yes (First approved July 2nd, 2015)
- Brand name: Orkambi
- Generic name: ivacaftor and lumacaftor
- Dosage form: Tablets
- 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 6 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 6 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), already FDA approved to treat a smaller subset of CF patients with different gene mutations.
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 tablets are taken every 12 hours (morning and evening) with fat-containing foods.
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 History and FDA Approval Process for Orkambi
Disclaimer: Every effort has been made to ensure that the information provided here is accurate, up-to-date and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. This information has been compiled for use by healthcare practitioners and consumers in the United States. The absence of a warning for a given drug or combination thereof in no way should be construed to indicate that the drug or combination is safe, effective or appropriate for any given patient. If you have questions about the substances you are taking, check with your doctor, nurse or pharmacist.