Rosane Charlab

TL;DR: Belinostat was administered intravenously at a dose of 1000 mg/m2 over 30 minutes once daily on days 1 to 5 of a 21-day cycle and showed overall response rate (ORR), the key secondary efficacy endpoint, and the median duration of response was 8.4 months.

TL;DR: The FDA thought process and data supporting accelerated approval based on PALOMA-1 that may be contingent upon verification and description of clinical benefit in the ongoing and fully accrued confirmatory trial PALOMa-2 are summarized.

TL;DR: The FDA review and data supporting rucaparib's accelerated approval was based primarily on efficacy data from 106 patients with BRCA mutation–associated ovarian cancer who had prior treatment with two or more chemotherapies and safety data from 377 patients with ovarian cancer treated with rucapsarib.

TL;DR: A selection of case studies from the first 5 years of the US Food and Drug Administration's voluntary exploratory data submission programme, which also involves collaboration with the European Medicines Agency, are discussed, and general lessons are highlighted.

TL;DR: The efficacy of ivosidenib was established on the basis of complete remission (CR) + CR with partial hematologic recovery (CRh) rate, duration of CR + CRh, and conversion from transfusion dependence to transfusion independence (TI) in Study AG120-C-001, a single-arm trial.