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Safety Evaluated in Patients Age 2 Through 5 Years Who Are Homozygous for the F508del-CFTR Mutation
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)
ALT, alanine aminotransaminase; AST, aspartate aminotransaminase; BMI, body mass index; CI, confidence interval; FEV1, forced expiratory volume in 1 second; LS, least squares; q12h, every 12 hours; ULN, upper limit of normal.
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:
References: 1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019. 2. Milla CE, Ratjen F, Marigowda G, et al. On behalf of the VX13-809-011 Part B Investigator Group. Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2017;195(7):912-920. 3. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00008(2); 2017. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01794; 2017. 5. Chilvers M, Owen CA, Marigowda G, et al. Safety and efficacy of lumacaftor/ivacaftor (LUMA/IVA) in patients aged ≥6 years with CF homozygous for F508del–a phase 3 extension study. Poster and abstract presented at: 31st Annual North American Cystic Fibrosis Conference; Indianapolis, Indiana; November 2-4, 2017. 6. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-20-00333; 2018. 7. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01799; 2017. 8. Ratjen F, Hug C, Marigowda G, et al. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017;5(7):557-567. 9. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01795(2); 2017. 10. Kent L, Reix P, Innes JA, et al. Lung clearance index: evidence for use in clinical trials in cystic fibrosis. J Cyst Fibros. 2014;13(2):123-138.
References:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019. 2. Milla CE, Ratjen F, Marigowda G, et al. On behalf of the VX13-809-011 Part B Investigator Group. Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2017;195(7):912-920. 3. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-88-00008(2); 2017. 4. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01794; 2017. 5. Chilvers M, Owen CA, Marigowda G, et al. Safety and efficacy of lumacaftor/ivacaftor (LUMA/IVA) in patients aged ≥6 years with CF homozygous for F508del–a phase 3 extension study. Poster and abstract presented at: 31st Annual North American Cystic Fibrosis Conference; Indianapolis, Indiana; November 2-4, 2017. 6. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-HQ-20-00333; 2018. 7. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01799; 2017. 8. Ratjen F, Hug C, Marigowda G, et al. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017;5(7):557-567. 9. Data on file. Vertex Pharmaceuticals Incorporated. Boston, MA. VXR-US-20-01795(2); 2017. 10. Kent L, Reix P, Innes JA, et al. Lung clearance index: evidence for use in clinical trials in cystic fibrosis. J Cyst Fibros. 2014;13(2):123-138.
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); 2019.
References:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019. 2. Wainwright CE, Elborn JS, Ramsey BW, et al. for the TRAFFIC and TRANSPORT Study Groups. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med. 2015;373(3):220-231.
Reference:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019.
References:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; June 2019. 2. Data on file. Vertex Pharmaceuticals Incorporated. Boston. MA. VXR-HQ-88-00201; 2018.
References:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019. 2. Data on file. Boston, MA. Vertex Pharmaceuticals Incorporated; REF-3538 (v1.0); 2019. 3. FDA U.S. Food & Drug Administration. Drugs@FDA: FDA-Approved Drugs. https://www.accessdata.fda.gov/scripts/cder/daf/. Accessed March 9, 2020 4. Xanax [prescribing information]. New York, NY: Pfizer; January 2017. 5. Klonopin [prescribing information]. South San Francisco, CA: Genentech, Inc.; October 2017. 6. Valium [prescribing information]. South San Francisco, CA: Genentech, Inc.; April 2017. 7. Kunze A, Huwyler J, Camenisch G, Poller B. Prediction of organic anion-transporting polypeptide 1B1- and 1B3-mediated hepatic uptake of statins based on transporter protein expression and activity data. Drug Metab Dispos. 2014;42(9):1514-1521.
Reference:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019.
Reference:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019.
Reference:
1. ORKAMBI [prescribing information]. Boston, MA: Vertex Pharmaceuticals Incorporated; July 2019