Drug updated on 4/17/2024
| Dosage Form | Tablet (oral; 150 mg); Granule (oral; 5.8 mg, 13.4 mg, 25 mg, 50 mg, 75 mg) |
| Drug Class | CFTR potentiators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.
Summary
- Ivacaftor (Kalydeco) is indicated for the treatment of cystic fibrosis in patients aged 1 month and older who have at least one mutation in the CFTR gene that responds to ivacaftor, based on clinical or in vitro assay data.
- Fifteen studies were reviewed, which provided comprehensive insights into ivacaftor's clinical performance within the broader CFTR modulator landscape.
- As part of triple therapy with elexacaftor and tezacaftor, ivacaftor demonstrates substantial improvements in lung function, quality of life, BMI across several studies, and shows a reduction in acute pulmonary exacerbations, signifying its potent role as a therapeutic regimen for cystic fibrosis.
- Dual therapy combinations such as lumacaftor-ivacaftor and tezacaftor-ivacaftor show meaningful improvements but with variations in safety profiles. The combination lumacaftor-ivacaftor was associated with early transient breathlessness and long-term increases in blood pressure, concerns not highlighted with tezacaftor-ivacaftor.
- Real-world evidence suggests that ivacaftor, alone or combined, is generally well-tolerated by patients, aligning adverse events observed during clinical trials, making it safe compared to other drugs used for treating cystic fibrosis.
- Efficacy varies across different genetic mutations, age groups, and the presence of specific comorbidities like Cystic Fibrosis Related Diabetes (CFRD). Significant benefits were noted, particularly in populations with one or two copies of the F508del mutation when treated with triple therapy, showing a favorable safety profile across various mutations.
- The impact on the pediatric population, especially those under years of age, is promising with notable improvements in lung function and growth parameters without significant safety concerns, highlighting the potential for early intervention in suitable candidates.
- Ivacaftor marks a significant leap in care by addressing the underlying genetic cause of the disease rather than just managing symptoms. These findings suggest a reevaluation of dual therapies in favor of triple therapy due to superior efficacy and manageable safety profiles, however, age restrictions and the precise genotypic profile of the patient are critical in therapy selection.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Kalydeco (ivacaftor) Prescribing Information. | 2023 | Vertex Pharmaceuticals Incorporated, Boston, MA |