FDA actions

The FDA has approved a new drug for the treatment of chronic myelogenous leukemia (CML). Omacetaxine mepesuccinate is a protein translation inhibitor that was originally identified in the 1970s as a potential treatment for CML as well as other hematologic conditions and even solid tumors. It was eventually dropped from development as the tyrosine kinase inhibitors (TKIs) became the mainstay of therapy. Emerging resistance to imatinib and other TKIs has led to renewed interest in the drug. It was recently approved for chronic, accelerated, or blast-phase Philadelphia-chromosome-positive CML that is resistant or in patients who are intolerant of other therapies including TKIs. Approval was based on a study of patients in chronic or accelerated-phase CML who had been treated with two or more TKIs. Omacetaxine is administered by subcutaneous injection. It is marketed by Teva Pharmaceuticals as Synribo. It joins Pfizer's bosutinib (Bosulif), which also was recently approved for the same indication.

The FDA has approved perampanel as adjunctive treatment for partial onset seizures in patients 12 years of age and older. The drug is the first in its class of noncompetitive AMPA receptor antagonists that are taken orally once daily. Approval was based on data from three Phase 3 studies of nearly 1500 patients with partial-onset seizures which found that perampanel, when used as an adjunctive therapy with other anti-seizure medications, significantly reduced seizure frequency. The drug comes with a boxed warning regarding serious neuropsychiatric events including agitation, aggression, anxiety, paranoia, euphoria, anger, and irritably. Perampanel is marketed by Eisai Inc. as Fycompa.