Drug updated on 4/17/2024
| Dosage Form | Tablet (oral; lumacaftor/ivacaftor: 100/125 mg, 200/125 mg); Granules (oral; lumacaftor/ivacaftor: 100/125 mg, 150/188 mg) |
| Drug Class | CFTR potentiators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of cystic fibrosis (CF) in patients age 2 years and older who are homozygous for the F508del mutation in the CFTR gene.
Summary
- Lumacaftor/ivacaftor (Orkambi) is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene.
- According to a systematic review, lumacaftor/ivacaftor has shown significant improvements in lung function, nutritional status, and clinical score among cystic fibrosis patients with F508del-CFTR homozygous mutation while maintaining an acceptable safety profile.
- The drug's efficacy varies depending on specific genetic mutations; it showed significant improvement when used by individuals with G551D or R117H mutations but had mixed effects on those with class II mutations taking ivacaftor along with lumacaftor.
- Despite its benefits, real-world studies indicate that discontinuation rates and adverse events may be higher than what was observed during clinical trials; respiratory-related adverse events were more frequent especially when using lumacaftor/ivacaftor combination therapy compared to other modulators like tezecaftar-ivacafter or elexcafter-tezecafter-ivafter combinations.
- A total of six systematic reviews/meta-analyses were reviewed which included randomized controlled trials investigating various aspects such as changes in forced expiratory volume (FEV), body mass index (BMI), Cystic Fibrosis Questionnaire-Revised scores, sweat chloride concentration levels etc., providing comprehensive insights into Orkambi's effectiveness and safety profiles across different patient populations based on their genotypes.
- While these documents provide valuable information about Orkambi’s potential benefits for certain subgroups within the cystic fibrosis population, they also highlight areas where further research is needed including long-term impact on nutritional status considering dietary intake factors as well as understanding patient characteristics associated with higher risk of certain adverse events related to this medication use.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Orkambi (lumacaftor and ivacaftor) Prescribing Information | 2022 | Vertex Pharmaceuticals Incorporated, Boston, MA |