Turalio First FDA-Approved Systemic Therapy for Tenosynovial Giant-Cell Tumor

Web Exclusives - FDA Updates

On August 2, 2019, the FDA approved pexidartinib (Turalio; Daiichi Sankyo) capsules, a kinase inhibitor, for adults with symptomatic tenosynovial giant-cell tumor (TGCT), which is associated with severe morbidity or functional limitations, who are not candidates for surgery. Pexidartinib is the first systemic therapy approved for patients with TGCT. The FDA granted pexidartinib a priority review and breakthrough therapy and orphan drug designations.

The FDA approval was based on the international, multicenter, randomized (1:1), double-blind, placebo-controlled clinical trial of 120 patients with TGCT that could not be removed by surgery. The primary end point was durable overall response rate (ORR), as determined by an independent review committee at week 25.

Patients were randomized in a 1:1 ratio to pexidartinib or to placebo. After 25 weeks, the ORR was 38% (95% confidence interval, 27-50), including 15% complete responses and 23% partial responses, with pexidartinib versus no responses with placebo (P <.0001). Overall, 22 patients who responded to pexidartinib therapy maintained the response for ≥6 months. In addition, 13 patients maintained the response for ≥12 months.

Patients with enough data had significant improvement in the range of motion of the affected joint at week 25 with pexidartinib versus placebo.

The most common side effects with pexidartinib were increased lactate dehydrogenase, increased aspartate aminotransferase, hair color changes, increased alanine aminotransferase, and increased cholesterol. Pexidartinib was approved with a boxed warning about the risk for fatal liver injury; the drug is only available through a Risk Evaluation and Mitigation Strategy program.

Related Items
November 11, 2019 — Oncology News & Updates
Web Exclusives published on November 11, 2019 in FDA Updates, In the News
FDA News
TOP - November 2019, Vol 12, No 4 published on November 7, 2019 in FDA Updates
Nubeqa New Oral Therapy FDA Approved for Nonmetastatic Prostate Cancer
Web Exclusives published on October 29, 2019 in FDA Updates
Rozlytrek Third FDA-Approved Tumor-Agnostic Drug and Second Treatment for Patients with NTRK Fusion Tumors
Web Exclusives published on October 29, 2019 in FDA Updates
October 28, 2019 – Oncology News & Updates
Web Exclusives published on October 28, 2019 in FDA Updates, In the News, Ovarian Cancer
Vincristine, Racial Disparities in Multiple Myeloma, and HER2 Metastatic Breast Cancer Are in the News
Web Exclusives published on October 22, 2019 in Breast Cancer, FDA Updates, In the News, Multiple Myeloma, Pediatric Cancer
FDA on In Vitro Diagnostics, Vaping; Cost of Novel Treatments
Web Exclusives published on October 15, 2019 in FDA Updates, In the News, NCCN
Darzalex Combination Receives Approval for First-Line Treatment of Patients with Multiple Myeloma Eligible for Transplant
Web Exclusives published on September 30, 2019 in FDA Updates, In the News, Multiple Myeloma
FDA News
TOP - August 2019, Vol 12, No 3 published on July 29, 2019 in FDA Updates
Keytruda Approved for Patients with Previously Treated Metastatic Small-Cell Lung Cancer
Yvette Florio Lane
Web Exclusives published on June 26, 2019 in FDA Updates, In the News
Last modified: October 29, 2019