Vanzacaftor | CAS#2374124-49-7 | CFTR protein modulator

MedKoo Cat#: 465969 | Name: Vanzacaftor

Featured

Description:

WARNING: This product is for research use only, not for human or veterinary use.

Vanzacaftor is a next-generation CFTR (cystic fibrosis transmembrane conductance regulator) modulator developed as a corrector for cystic fibrosis treatment. It is designed to enhance CFTR protein folding and trafficking, particularly in combination with other modulators like deutivacaftor and tezacaftor. In preclinical and clinical studies, vanzacaftor has demonstrated high potency in improving chloride transport in CFTR-mutant cells, particularly those with the common F508del mutation. Studies have reported significant increases in CFTR function, with EC50 values in the low nanomolar range, indicating strong bioactivity.

Chemical Structure

Vanzacaftor

CAS#2374124-49-7

Theoretical Analysis

MedKoo Cat#: 465969

Name: Vanzacaftor

CAS#: 2374124-49-7

Chemical Formula: C33H41N7O4S

Exact Mass: 631.2941

Molecular Weight: 631.80

Elemental Analysis: C, 62.74; H, 6.54; N, 15.52; O, 10.13; S, 5.07

Price and Availability

Size	Price	Availability	Quantity
5mg	USD 750.00	2 Weeks
25mg	USD 1,650.00	2 Weeks

Bulk Inquiry

Buy Now

Add to Cart

Related CAS #

No Data

Synonym

Vanzacaftor; VX-121; VX 121; VX121;

IUPAC/Chemical Name

(13S)-26-(3-(2-(dispiro[2.0.24.13]heptan-7-yl)ethoxy)-1H-pyrazol-1-yl)-13,15,15-trimethyl-5-thia-4,7-diaza-2(2,3),6(2,6)-dipyridina-1(1,3)-pyrrolidinacyclodecaphan-3-one 5,5-dioxide

InChi Key

BUJTZBZUCRYTIH-HKBQPEDESA-N

InChi Code

InChI=1S/C33H41N7O4S/c1-30(2)20-31(3)12-5-17-34-24-6-4-7-27(35-24)45(42,43)38-29(41)22-8-9-25(36-28(22)39(30)21-31)40-18-10-26(37-40)44-19-11-23-32(13-14-32)33(23)15-16-33/h4,6-10,18,23H,5,11-17,19-21H2,1-3H3,(H,34,35)(H,38,41)/t31-/m0/s1

SMILES Code

O=C(NS1(=O)=O)C2=CC=C(N=C2N3C(C)(C)C[C@@](CCCNC4=NC1=CC=C4)(C)C3)N5N=C(OCCC6C7(CC7)C68CC8)C=C5

Appearance

Solid powder

Purity

>98% (or refer to the Certificate of Analysis)

Shipping Condition

Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition

Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility

To be determined

Shelf Life

>2 years if stored properly

Drug Formulation

To be determined

Stock Solution Storage

0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code

2934.99.9001

More Info

Instruction

View handling instruction

Preparing Stock Solutions

The following data is based on the product molecular weight 631.80 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Concentration / Solvent Volume / Mass	1 mg	5 mg	10 mg
1 mM	1.15 mL	5.76 mL	11.51 mL
5 mM	0.23 mL	1.15 mL	2.3 mL
10 mM	0.12 mL	0.58 mL	1.15 mL
50 mM	0.02 mL	0.12 mL	0.23 mL

1: Correction. Med Lett Drugs Ther. 2025 Mar 31;67(1725):e56. doi: 10.58347/tml.2025.1725d. PMID: 40152724. 2: Iftikhar IH, Rao ST, Nadama R, Janahi I, BaHammam AS. Comparative Efficacy of CFTR Modulators: A Network Meta-analysis. Lung. 2025 Mar 18;203(1):49. doi: 10.1007/s00408-025-00802-w. PMID: 40102290. 3: Drugs and Lactation Database (LactMed®) [Internet]. Bethesda (MD): National Institute of Child Health and Human Development; 2006–. Vanzacaftor, Tezacaftor and Deutivacaftor. 2025 Mar 15. PMID: 39836862. 4: Vanzacaftor, tezacaftor, and deutivacaftor (Alyftrek) for cystic fibrosis. Med Lett Drugs Ther. 2025 Mar 17;67(1724):41-43. doi: 10.58347/tml.2025.1724a. PMID: 40053374. 5: Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016/S2213-2600(24)00407-7. Epub 2025 Jan 2. Erratum in: Lancet Respir Med. 2025 Mar;13(3):e19. doi: 10.1016/S2213-2600(25)00010-4. PMID: 39756425. 6: Keating C, Yonker LM, Vermeulen F, Prais D, Linnemann RW, Trimble A, Kotsimbos T, Mermis J, Braun AT, O'Carroll M, Sutharsan S, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Floreth T, Michelson P, Sosnay PR, Nair N, Zahigian R, Martin H, Ahluwalia N, Lam A, Horsley A; VX20-121-102 Study Group; VX20-121-103 Study Group. Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. Lancet Respir Med. 2025 Mar;13(3):256-271. doi: 10.1016/S2213-2600(24)00411-9. Epub 2025 Jan 2. Erratum in: Lancet Respir Med. 2025 Mar;13(3):e19. doi: 10.1016/S2213-2600(25)00011-6. PMID: 39756424. 7: Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23. PMID: 36842446.