New FDA breakthrough-drug category - Implications for patients [9]

摘要

The article by Darrow et al. summarizes prior government efforts to expedite the availability of new therapeutics and discusses the implications of the breakthrough-therapy designation. It is worth clarifying that gemtuzumab ozogamicin was not approved for the treatment of pediatric leukemia. Three trials evaluated the efficacy and safety of the single agent gemtuzumab ozogamicin. The population for the initial report included 142 patients with a median age of 61 years who had a first relapse of acute myeloid leukemia (AML). A total of 30% of the patients had remission. The FDA granted approval for gemtuzumab ozogamicin in the treatment of patients with a first relapse of CD33-positive AML who were 60 years of age or older and who were not considered candidates for cytotoxic chemotherapy.