Elexacaftor + Tezacaftor + Ivacaftor
Indications
Elexacaftor + Tezacaftor + Ivacaftor is used for:
Cystic Fibrosis
Adult Dose
Cystic Fibrosis
Indicated for cystic fibrosis in patients who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population
2 fixed-dose tablets (elexacaftor 100 mg, tezacaftor 50 mg, and ivacaftor 75 mg) PO qAM and 1 ivacaftor 150-mg tablet PO qPM; ~12 hr apart
Hepatic impairment
Mild (Child-Pugh A): No dosage adjustment required
Moderate (Child-Pugh B): Not recommended unless benefit exceeds risk; reduce dose by omitting evening ivacaftor 150-mg tab; monitor liver function tests
Severe (Child-Pugh C): Do not use
Child Dose
Cystic Fibrosis
Indicated for cystic fibrosis in children aged ?12 years who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population
2 fixed-dose tablets (elexacaftor 100 mg, tezacaftor 50 mg, and ivacaftor 75 mg) PO qAM and 1 ivacaftor 150-mg tablet PO qPM; ~12 hr apart
Renal Dose
Renal impairment
Mild or moderate (eGFR 30 to <90 mL/min/1.73 m2): No dosage adjustment required
Severe (eGFR <30 mL/min/1.73 m2) or ESRD: Use caution
Administration
Swallow tablets whole; do not chew, crush, or split
Take with fat-containing food (eg, meals or snacks prepared with butter or oils or those containing eggs, cheeses, nuts, whole milk, or meats)
Contra Indications
Precautions
Elevated liver transaminases and bilirubin levels observed; measure levels before initiating, q3Months during first year, and annually thereafter; consider more frequent monitoring for those with history of hepatic disease; interrupt dosing for significant elevations
Noncongenital lens opacities reported with ivacaftor-containing regimens; other risk factors were present in some cases (eg, corticosteroid use, radiation exposure); a possible risk attributable to treatment with ivacaftor cannot be excluded
Pregnancy-Lactation
Pregnancy
Human data are limited and incomplete from clinical trials on the use of elexacaftor/tezacaftor/ivacaftor or its individual components in pregnant women to inform a drug-associated risk
Although there are no animal reproduction studies with the concomitant administration of elexacaftor, tezacaftor, and ivacaftor, separate reproductive and developmental studies were conducted with each active component in pregnant rats and rabbits
Animal data
In animal embryofetal development studies, oral administration of elexacaftor to pregnant rats and rabbits during organogenesis demonstrated no teratogenicity or adverse developmental effects at doses that produced maternal exposures up to ~2 times the exposure at the maximum recommended human dose (MRHD) in rats and 4 times the MRHD in rabbits
Lactation
No data are available regarding the presence of elexacaftor, tezacaftor, or ivacaftor in human milk, the effects on the breastfed infant, or the effects on milk production.
Elexacaftor, tezacaftor, and ivacaftor are excreted into the milk of lactating rats
Interactions
CYP3A inhibitors or inducers
Coadministration with moderate or strong CYP3A inhibitors increases systemic exposure of elexacaftor/tezacaftor/ivacaftor; dosage adjustment is required if coadministered
Coadministration with strong CYP3A inducers is not recommended; ivacaftor systemic exposure is significantly decreased and exposure to elexacaftor and tezacaftor are expected to decrease if coadministered with a strong CYP3A inducer
Potential for elexacaftor/tezacaftor/ivacaftor to affect other drugs
Ivacaftor may inhibit CYP2C9; monitor INR if coadministered with warfarin; caution with other CYP2C9 substrates (eg, glimepiride, glipizide)
Coadministration of ivacaftor or tezacaftor/ivacaftor with digoxin, a sensitive P-gp substrate, increased digoxin AUC by 1.3-fold, consistent with weak inhibition of P-gp by ivacaftor
Elexacaftor and its active metabolite (M23-ELX) inhibit uptake by OATP1B1 and OATP1B3 in vitro; coadministration may increase exposures of drugs that are substrates of these transporters (eg, statins, glyburide, nateglinide, repaglinide)
Adverse Effects
Side effects of Elexacaftor + Tezacaftor + Ivacaftor :
>10%
Headache (17%)
Upper respiratory tract infection (16%)
Abdominal pain (14%)
Diarrhea (13%)
1-10%
Rash (10%)
Increased ALT (10%)
Increased AST (9%)
Increased BUN (9%)
Nasal congestion (9%)
Rhinorrhea (8%)
Rhinitis (7%)
Influenza (7%)
Sinusitis (5%)
Increased bilirubin (5%)
2 to <5%
Flatulence
Abdominal distension
Conjunctivitis
Pharyngitis
Respiratory tract infection
Tonsillitis
Urinary tract infection
Increased C-reactive protein
Hypoglycemia
Dizziness
Dysmenorrhea
Acne
Eczema
Pruritus
Mechanism of Action
Elexacaftor and tezacaftor bind to different sites on the cystic fibrosis transmembrane conductance regulator (CFTR) protein and have an additive effect in facilitating the cellular processing and trafficking of F508del-CFTR to increase the amount of CFTR protein delivered to the cell surface compared with either molecule alone
Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface