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Trial 6 was a 24-week, Phase 3, open-label study assessing the safety, tolerability, and pharmacokinetics of ORKAMBI in 60 patients age 2 through 5 years with CF who were homozygous for the F508del-CFTR mutation. Patients received ORKAMBI granules (if <14 kg: lumacaftor 100 mg/ivacaftor 125 mg, or if ≥14 kg: lumacaftor 150 mg/ivacaftor 188 mg) mixed with 1 teaspoon (5 mL) of soft food or liquid and administered orally every 12 hours with fat-containing food. All patients continued to take their prescribed CF therapies (including during the 2-week washout period). The primary endpoint was safety and tolerability up to Week 24.1,2
Download the Clinical Brochure including information from Trial 6 in patients age 2 through 5 years.
Adverse Event (Preferred Term) |
ORKAMBI Total, N=60 n(%) |
---|---|
Patients with any adverse event of special interest of respiratory symptoms | 3 (5.0) |
Dyspnea | 3 (5.0) |
Respiration abnormal | 1 (1.7) |
Elevated ALT or AST | ORKAMBI Total, N=60 n(%) |
---|---|
ALT (ULN) | |
>3 x to ≤5 x ULN | 2 (3.3) |
>5 x to ≤8 x ULN | 2 (3.3) |
>8 x ULN | 5 (8.3) |
AST (ULN) | |
>3 x to ≤5 x ULN | 3 (5.0) |
>5 x to ≤8 x ULN | 1 (1.7) |
>8 x ULN | 1 (1.7) |
ALT or AST | |
(ALT >3 x ULN) or (AST >3 x ULN) | 9 (15.0) |
(ALT >5 x ULN) or (AST >5 x ULN) | 7 (11.7) |
(ALT >8 x ULN) or (AST >8 x ULN) | 5 (8.3) |
ORKAMBI® (lumacaftor/ivacaftor) is a combination of lumacaftor and ivacaftor indicated 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. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene.
The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation.
Substrates of CYP3A
Strong CYP3A Inducers
Click here to access full Prescribing Information.
References:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019. 2. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00178; 2018. 3. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00180; 2018. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00179(1); 2018.