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Alyftrek (VANZACAFTOR)
Alyftrek potentiates the CFTR protein to facilitate chloride ion flow into cells.
Alyftrek (vanzacaftor) is a small molecule drug developed by Vertex Pharmaceuticals Inc, classified as a Cystic Fibrosis Transmembrane Conductance Regulator Potentiator. It is used to treat cystic fibrosis, a genetic disorder that affects the lungs and digestive system. Alyftrek works by potentiating the CFTR protein, allowing chloride ions to flow into cells and thin mucus, making it easier to expel. It was FDA-approved in 2026 and is currently patented. Key safety considerations include potential interactions with other medications and monitoring for liver enzyme elevations.
At a glance
| Generic name | VANZACAFTOR |
|---|---|
| Sponsor | Vertex Pharms Inc |
| Drug class | Cystic Fibrosis Transmembrane Conductance Regulator Potentiator [EPC] |
| Modality | Small molecule |
| Therapeutic area | Respiratory |
| Phase | FDA-approved |
| First approval | 2026 |
Mechanism of action
Vanzacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del- CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Deutivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of vanzacaftor, tezacaftor and deutivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increased CFTR activity as measured both by CFTR mediated chloride transport in vitro and by sweat chloride in patients with CF. CFTR Chloride Transport Assay in Fischer Rat Thyroid Cells Expressing Mutant CFTR Protein Effects of vanzacaftor/tezacaftor/deutivacaftor on chloride transport for mutant CFTR protein was determined in Ussing chamber electrophysiology studies using a panel of Fischer Rat Thyroid
Approved indications
- cystic fibrosis
- cystic fibrosis
Boxed warnings
- WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE Elevated transaminases have been observed in patients treated with ALYFTREK. Cases of serious and potentially fatal drug-induced liver injury and liver failure were reported in patients who were taking a fixed-dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of elexacaftor/tezacaftor/ivacaftor [see Warnings and Precautions (5.1) and Adverse Reactions (6) ]. Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK, every month during the first 6 months of treatment, then every 3 months for the next 12 months, then at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or elevated liver function tests at baseline [see Dosage and Administration (2.1) , Warnings and Precautions (5.1) , Adverse Reactions (6) , and Use in Specific Populations (8.7) ] . Interrupt ALYFTREK for significant elevations in liver function tests or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If abnormalities resolve, resume treatment only if the benefit is expected to outweigh the risk. Closer monitoring is advised after resuming ALYFTREK [see Warnings and Precautions (5.1) ] . ALYFTREK should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B) and should only be considered when there is a clear medical need, and the benefit outweighs the risk. If used, monitor patients closely [see Dosage and Administration (2.4) , Warnings and Precautions (5.1) , Adverse Reactions (6) , Use in Specific Populations (8.7) , and Clinical Pharmacology (12.3) ] . WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE See full prescribing information for complete boxed warning. Elevated transaminases have been observed in patients treated with ALYFTREK ( 5.1 , 6 ). Cases of serious and potentially fatal drug-induced liver injury and liver failure leading to transplantation and death were reported in patients who were taking ELX/TEZ/IVA, a drug containing the same or similar active ingredients as ALYFTREK ( 5.1 ). Assess liver function tests (ALT, AST, alkaline phosphatase, bilirubin) in all patients prior to initiating ALYFTREK, every month for first 6 months, every 3 months for next 12 months, then at least annually ( 2.1 , 5.1 ). Interrupt ALYFTREK for significant elevations in LFTs or signs or symptoms of liver injury. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve ( 5.1 ). Resume ALYFTREK if abnormalities resolve and only if the benefit is expected to outweigh the risk ( 5.1 ). ALYFTREK should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B) ( 2.4 , 5.1 , 8.7 , 12.3 ).
Common side effects
- Cough
- Nasopharyngitis
- Upper respiratory tract infection
- Headache
- Oropharyngeal pain
- Influenza
- Fatigue
- ALT increased
- Rash
- AST increased
- Sinus congestion
- Infective pulmonary exacerbation of CF
Drug interactions
- Strong or moderate CYP3A inducers
- Strong or moderate CYP3A inhibitors
- Grapefruit
- Vanzacaftor, tezacaftor, and deutivacaftor
- Vanzacaftor, tezacaftor, and deutivacaftor
- Vanzacaftor, tezacaftor, and deutivacaftor
- Grapefruit juice
- P-glycoprotein (P-gp) substrates
- Breast Cancer Resistance Protein (BCRP) substrates
- CYP2C9 substrates
- Warfarin
- Ciprofloxacin
Key clinical trials
- A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) (PHASE3)
- A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation (PHASE3)
- Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis (PHASE1)
- Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants (PHASE1)
- Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
- Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older (PHASE3)
- Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
- A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA) (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Alyftrek CI brief — competitive landscape report
- Alyftrek updates RSS · CI watch RSS
- Vertex Pharms Inc portfolio CI