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VOXELOTOR
CAS NO.: 1446321-46-5
Chemical Formula: C19H19N3O3
Molecular Weight: 337.4000
DMF&GMP status: Please contact us for more details.
Description:
Voxelotor is an orally bioavailable modulator and stabilizer of sickle cell hemoglobin (HbS), with potential use in the treatment of sickle cell disease (SCD). Upon administration, voxelotor targets and covalently binds to the N-terminal valine of the alpha chain of HbS. This stabilizes HbS, thereby improving oxygen binding affinity. The binding of voxelotor to HbS prevents HbS polymerization, reduces sickling, decreases red blood cell (RBC) damage and increases the half-life of RBCs. This improves blood flow and decreases hemolytic anemia.

Voxelotor is a novel hemoglobin S polymerization inhibitor for the treatment of sickle cell disease. This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India. Sickle cell disease can lead to excruciating pain, stroke, infection, and various other complications arising from the blockage of blood vessels. Voxelotor was granted accelerated FDA approval on November 25 2019, as it likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition to 20 million others worldwide. It was developed by Global Blood Therapeutics, Inc. and is unique from other drugs used to treat sickle cell anemia, such as [hydroxyurea], [L-glutamine], and [crizanlizumab] due to its novel mechanism of action.

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Approvals
Active Ingredient Dosage Form; Route Strength Proprietary Name Applicant
VOXELOTORTABLET;ORAL500MGOXBRYTAGLOBAL BLOOD THERAPEUTICS INC
Patent Data
Patent No. Patent Expiration Drug Substance Claim Drug Product Claim Patent Use Code
N.A.N.A.N.A.N.A.N.A.
Exclusive Data
Exclusivity Code Exclusivity Expiration
NCE 11/25/2024
ODE-281 11/25/2026