2374124-49-7

Product Details:

CasNo： 2374124-49-7

Molecular Formula： C32H39N7O4S

Appearance： Off-white to pale yellow powder

Delivery Time： 2 weeks after order

Packing： Aluminium bag

Purity： 99%

Vanzacaftor (VX‑121) is a next‑generation CFTR corrector developed by Vertex Pharmaceuticals, designed to treat cystic fibrosis (CF). It is the core component of the triple‑combination oral therapy Alyftrek® (vanzacaftor/tezacaftor/deutivacaftor), approved by the U.S. FDA on December 20, 2024, for patients aged 6 years and older.

1. Basic Information

• Generic Name: Vanzacaftor
• Synonym: VX‑121
• Drug Class: Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Corrector
• Combination Product: Alyftrek® (Vanzacaftor + Tezacaftor + Deutivacaftor)
• Developer: Vertex Pharmaceuticals
• Approval: U.S. FDA (Dec 20, 2024); Swissmedic (Oct 15, 2025)
• Indication: For patients 6 years and older with cystic fibrosis who have at least one F508del mutation or another CFTR‑responsive mutation.

2. Mechanism of Action

Vanzacaftor works synergistically with tezacaftor (another corrector) and deutivacaftor (a CFTR potentiator) to restore defective CFTR protein functionNLM:

1. Vanzacaftor + Tezacaftor (Correctors): Promote proper folding, processing, and trafficking of mutant CFTR proteins (e.g., F508del‑CFTR), increasing the number of functional CFTR channels at the cell surface.
2. Deutivacaftor (Potentiator): Enhances the opening probability of surface‑localized CFTR channels, boosting chloride ion transport efficiencyNLM.
3. Net Effect: Reduces thick mucus buildup in the lungs and other organs, alleviating CF symptomsNLM.

3. Dosage Forms & Administration (Alyftrek®)

Alyftrek® is administered once daily orally with fat‑containing food to optimize absorptionNLM. Two strengths are available:

• Alyftrek 4/20/50 mg (purple round): Vanzacaftor 4 mg + Tezacaftor 20 mg + Deutivacaftor 50 mg. For 6 to <12 years, <40 kg: 3 tablets daily.
• Alyftrek 10/50/125 mg (purple oval): Vanzacaftor 10 mg + Tezacaftor 50 mg + Deutivacaftor 125 mg. For 6 to <12 years, ≥40 kg or ≥12 years: 2 tablets daily.

4. Clinical Benefits

• Improved Lung Function: Significant increases in FEV₁ (forced expiratory volume in 1 second).
• Reduced Exacerbations: Lower frequency of pulmonary infections and hospitalizations.
• Enhanced Quality of Life: Alleviates cough, dyspnea, and other respiratory symptoms.
• Nutritional Improvement: Supports pancreatic function and weight gain.
• Biomarker Response: Reduces sweat chloride levels, a key indicator of therapeutic success.

5. Safety Profile

• Common Adverse Reactions: Headache, diarrhea, nausea, elevated transaminases, nasopharyngitis, rashNLM.
• Boxed Warning: Hepatotoxicity (elevated liver enzymes, severe liver injury/failure). Liver function monitoring is required before and during treatmentNLM.
• Drug Interactions: Avoid co‑administration with strong CYP3A inducers (e.g., rifampin).
• Special Populations: Safety not established in patients <6 years; use in pregnancy/lactation only if benefits outweigh risks.

6. Comparison with Trikafta®

• Alyftrek®: Vanzacaftor + Tezacaftor + Deutivacaftor (next‑generation; deutivacaftor is a deuterated analog of ivacaftor with improved pharmacokinetics).
• Trikafta®: Elexacaftor + Tezacaftor + Ivacaftor (first‑generation triple combination).
• Advantage: Alyftrek® offers a favorable patent landscape and commercial potential, potentially improving patient access.

7. Summary

Vanzacaftor is a pivotal next‑generation CFTR corrector that, in combination with tezacaftor and deutivacaftor as Alyftrek®, provides a safe, effective, once‑daily treatment option for 6 years and older patients with specific CFTR mutations. It addresses the underlying cause of cystic fibrosis, delivering meaningful improvements in lung function, symptom control, and quality of life.