FDA Approves Pimicotinib for Symptomatic Tenosynovial Giant Cell Tumor

The FDA has approved pimicotinib (Pimjetmi), a kinase inhibitor, for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity. The recommended dose is 50 mg orally once daily, with or without food.¹

The approval is supported by the international, double-blind, phase 3 MANEUVER study (NCT05804045), in which patients were randomly assigned 2:1 to pimicotinib at 50 mg once daily or placebo, with randomization stratified by geographic region (China vs non-China). Eligible patients had a confirmed diagnosis of symptomatic TGCT with measurable disease, including at least 1 lesion of at least 2 cm.

Efficacy Results From MANEUVER

The FDA notice did not report efficacy data. In part 1 of the study, published in The Lancet, 94 patients were randomly assigned to pimicotinib (n = 63) or placebo (n = 31).² The primary end point was objective response rate (ORR) at week 25 by blinded independent review committee per RECIST v1.1 in the intention-to-treat population.²

At week 25, the ORR was 54.0% (n = 34/63) with pimicotinib vs 3.2% (n = 1/31) with placebo, an absolute difference of 51% (95% CI, 33%-63%; P < .0001).^2,4 With a median follow-up of 14.3 months, the ORR among patients treated with pimicotinib from the start of the study increased to 76.2% (95% CI, 63.8%-86.0%) per RECIST v1.1, according to data presented at the 2025 ESMO Congress.³

Safety Profile

The most common adverse reactions (>20%) with pimicotinib were pruritus, rash, face edema, periorbital edema, fatigue, nausea, headache, and peripheral edema.¹ The most common laboratory abnormalities (>30%) were increased creatine phosphokinase, lactate dehydrogenase, aspartate aminotransferase, lipase, cholesterol, amylase, and alanine aminotransferase.

Warnings and precautions include hepatotoxicity, embryo-fetal toxicity, and increased serum creatinine without an effect on renal function.

In the Lancet report, the only grade 3 or 4 treatment-emergent adverse event (TEAE) occurring in more than 10% of patients treated with pimicotinib was increased blood creatine phosphokinase, reported in 8 of 63 patients (13%).² In part 1, TEAEs led to treatment discontinuation in 1 patient (1.6%) and dose reduction in 5 patients (7.9%) in the pimicotinib arm.⁴

Regulatory Background

The application was granted fast track and breakthrough therapy designations and was reviewed under Project Orbis, with concurrent review by regulators in Australia, Canada, Israel, Switzerland, and the United Kingdom; the European Medicines Agency participated as an observer.¹ Reviews by other agencies may still be in progress.

For context, the FDA accepted the new drug application for pimicotinib in January 2026, and China's National Medical Products Administration approved the agent for symptomatic TGCT in December 2025.⁶ Pexidartinib (Turalio) and vimseltinib (Romvimza) are the other FDA-approved colony-stimulating factor 1 receptor inhibitors for TGCT.

FAQs

What did the FDA approve?

The FDA approved pimicotinib (Pimjetmi) for adults with symptomatic TGCT for which surgical resection may cause worsening functional limitation or severe morbidity.

What is the recommended dose of pimicotinib?

The recommended dose is 50 mg orally once daily, with or without food.

What data supported the approval?

The approval is supported by the phase 3 MANEUVER study (NCT05804045), which randomly assigned patients 2:1 to pimicotinib or placebo. At week 25, the objective response rate was 54.0% (34 of 63) with pimicotinib vs 3.2% (1 of 31) with placebo. With a median follow-up of 14.3 months, the rate among patients treated with pimicotinib from the start rose to 76.2%.

What are the most common adverse reactions?

Adverse reactions in more than 20% of patients were pruritus, rash, face edema, periorbital edema, fatigue, nausea, headache, and peripheral edema.

Are there other FDA-approved CSF-1R inhibitors for TGCT?

Yes. Pexidartinib (Turalio) and vimseltinib (Romvimza) are also FDA approved for TGCT.

REFERENCES

1. FDA approves pimicotinib for symptomatic tenosynovial giant cell tumor. News release. FDA. November 10, 2026. Accessed November 10, 2026. https://tinyurl.com/thz9wb9w

2. Xu H, Niu X, Ravi V, et al. Pimicotinib versus placebo for tenosynovial giant cell tumour (MANEUVER): an international, randomised, placebo-controlled, phase 3 trial. Lancet. 2026;407(10533):1072-1083. doi:10.1016/S0140-6736(25)02602-9

3. Niu X, Ravi V, Broto JM, et al. Extended efficacy and safety from the phase 3 MANEUVER trial of pimicotinib in patients with tenosynovial giant cell tumour. Presented at: 2025 ESMO Congress; October 17-21, 2025; Berlin, Germany. Abstract 2690.

4. Pimicotinib significantly improved outcomes for patients with tenosynovial giant cell tumor in a global phase III trial. News release. Abbisko. November 11, 2024. Accessed November 10, 2026. https://tinyurl.com/245uhhh2

5. FDA accepts new drug application for pimicotinib for the treatment of tenosynovial giant cell tumor. News release. EMD Serono. January 12, 2026. Accessed November 10, 2026. https://www.businesswire.com/news/home/20260108289260/en/