U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Status:
Designated
Source:
FDA ORPHAN DRUG:71792
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)


Isobutyramide (VX-366) is an orally available branched chain amide that may offer an alternative to current treatments for beta-hemoglobinopathy. Isobutyramide has been shown to increase fetal hemoglobin (HbF) in patients with beta-hemoglobinopathies. Isobutyramide was originated at the Children's Hospital Oakland Research Institute which, in 1993, granted exclusive worldwide rights for the agent to Vertex. However, development of Isobutyramide has been discontinued for sickle cell anaemia and thalassaemia.