fda-approvals-june-2019

FDA Approves Three Agents for Cancer Indications

  • First Ever Treatment for Tenosynovial Giant Cell Tumor. The United States Food and Drug Administration (FDA) granted approval to pexidartinib (Turalio™, Daiichi-Sankyo) for treatment of patients with symptomatic tenosynovial giant cell tumor (TGCT) with severe morbidity or functional limitations who cannot be treated with surgery. The approval was based on the phase III ENLIVEN study, in which pexidartinib resulted in a significant improvement in tumor response rate compared to placebo (38% vs 0%; P < .0001). A total of 15% of patients treated with pexidartinib achieved a complete response. Pexidartinib will carry a boxed warning for risk of serious and potentially fatal hepatotoxicity.
  • Entrectinib, a Tumor Agnostic Targeted Therapy. The TRK inhibitor entrectinib (Rozlytrek®, Genentech) received approval for treatment of solid tumors with a neurotrophic tyrosine receptor kinase (NTRK) gene fusion in patients who are 12 years of age or older, have advanced disease, and have progressed following treatment or have no satisfactory standard therapy. It is the second TRK inhibitor approved for a tumor agnostic indication, following the approval of larotrectinib in 2018. It was also simultaneously approved for treatment of metastatic, ROS1-positive non-small cell lung cancer (NSCLC). Approval was based on results from a dataset of four early-phase trials, which included 54 patients with advanced NTRK-positive solid tumors and 53 patients with ROS1 gene fusions. Entrectinib was associated with an overall response rate (ORR) of 57% in patients with NTRK-positive tumors and 78% in patients with ROS1-positive NSCLC.
  • Fedratinib. The JAK-2 inhibitor fedratinib (Inrebic®, Impact Biomedicines) was approved for treatment of patients with intermediate-2 or high-risk primary or secondary myelofibrosis (MF). This approval was based on reanalyzed results from the JAKARTA-2 trial, which compared fedratinib to placebo in patients with MF who had failed prior treatment with ruxolitinib. On this study, significantly more patients treated with fedratinib compared to placebo achieved a ³ 35% reduction in spleen volume (37% vs 1%; P < .0001). Fedratinib will carry a boxed warning regarding the risk of serious and fatal encephalopathy.

FDA-August-2019

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