FDA Approves Pimicotinib for Symptomatic Tenosynovial Giant Cell Tumor
Clinical Summary:
- Based on results from the phase 3 MANEUVER trial, the FDA has approved pimicotinib for adults with symptomatic tenosynovial giant cell tumor for whom surgery may result in worsening functional limitation or severe morbidity.
- In this randomized, double-blind study, pimicotinib was evaluated against placebo, with key safety considerations including hepatotoxicity, edema, and laboratory abnormalities.
- This approval provides a new systemic treatment option for patients with symptomatic tenosynovial giant cell tumor who may not be appropriate candidates for surgical resection.
On November 10, 2026, the US Food and Drug Administration (FDA) approved pimicotinib (Pimjetmi; EMD Serono, Inc), an oral colony stimulating factor-1 receptor (CSF-1R) inhibitor, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) for whom surgical resection may result in worsening functional limitation or severe morbidity.
This approval is based on primary and longer-term results from the phase 3 MANEUVER trial. This international, randomized, double-blind, placebo-controlled trial enrolled patients with symptomatic, measurable TGCT, including at least 1 lesion measuring ≥2 cm, and whom surgical resection could result in dysfunction or serious complication. Patients were randomized 2:1 to receive 50 mg pimicotinib orally once daily or placebo, with randomization stratified by geographic region.
Pimicotinib demonstrated a statistically significant improvement in objective response rate (ORR) at week 25 compared with placebo (56% vs 3% for placebo; P < .0001). In addition, pimicotinib showed clinically meaningful and statistically significant improvements versus placebo across secondary quality of life end points, improving relative range of motion (P = .0003), physical functioning (P = .0074), and pain (P < .0001).
Initial results were presented at the 2025 ASCO Annual Meeting with longer-term follow-up results (median, 14.3 months) presented at the 2025 ESMO Congress. The follow-up results showed that 77% of patients who had a response at 25 weeks maintained clinical benefit after an additional 12 months.
In MANEUVER, the most common adverse reactions occurring in more than 20% of patients were pruritus, rash, facial edema, periorbital edema, fatigue, nausea, headache, and peripheral edema. The most common laboratory abnormalities occurring in more than 30% of patients included increased creatine phosphokinase, lactate dehydrogenase, aspartate aminotransferase, lipase, cholesterol, amylase, and alanine aminotransferase.
The recommended dose of pimicotinib is 50 mg orally once daily, with or without food. Prescribing information includes warnings and precautions for hepatotoxicity, embryo-fetal toxicity, and increased serum creatinine without an effect on renal function.
Sources:
- US Food and Drug Administration. FDA approves pimicotinib for symptomatic tenosynovial giant cell tumor. Accessed on: November 10, 2026. https://www.fda.gov/drugs/cancer-approvals/fda-approves-pimicotinib-symptomatic-tenosynovial-giant-cell-tumor
- Business Wire. FDA Approves PIMJETMI® (pimicotinib) for Patients with Tenosynovial Giant Cell Tumor. Accessed on: November 10, 2026. https://www.businesswire.com/news/home/20261029670739/en/FDA-Approves-PIMJETMI-pimicotinib-for-Patients-with-Tenosynovial-Giant-Cell-Tumor
