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FDA OKs first in vitro route to expanded approval

In vitro data can be used to accelerate the approval of drugs that target specific disease-causing mutations for additional subpopulations of patients with rare diseases such as cystic fibrosis. Katie Kingwell In May, the FDA granted expanded approval to Vertex Pharmaceuticals’ cystic fibrosis (CF) drug ivacaftor on the basis of in vitro data. The change adds 23 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the existing 10 mutations previously covered by the drug label, representing a…

FDA approves first-in-class cancer metabolism drug

The FDA approved Agios’ and Celgene’s enasidenib for acute myeloid leukaemia (AML), validating metabolism-modulating drugs as a means of killing cancer cells. Enasidenib (formerly AG-221) is a first-in-class inhibitor of mutated isocitrate dehydro genase 2 (IDH2). The IDH enzymes normally metabolize isocitrate into α-ketoglutarate. When they are mutated in cancers, they also convert α-ketoglutarate into 2-hydroxyglutarate, an oncometabolite that causes cell differentiation defects by impairing histone demethylation. In clinical trials of enasidenib, 23% of treated patients had complete responses or complete…