FDA Approves Pimicotinib for Tenosynovial Giant Cell Tumor

On November 10, 2026, the FDA approved pimicotinib (Pimjetmi; EMD Serono), a once-daily oral kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity. The approval adds a third colony-stimulating factor-1 receptor (CSF-1R)–targeted therapy for a rare, locally aggressive tumor that largely affects otherwise healthy adults.^1,2

Unmet Need in TGCT

TGCT has an estimated annual global incidence of 43 cases per 1 million person-years. The disease can cause irreversible joint and bone damage, and patients commonly report pain, swelling, stiffness, and reduced range of motion. Surgery has been the primary treatment, but systemic therapy may be the only suitable option for patients with unresectable disease.²

The FDA granted pimicotinib fast track and breakthrough therapy designations. Pimicotinib, which is developed by Abbisko Therapeutics, was first approved in China in December 2025.^1,3

MANEUVER Trial Results

The approval is based on MANEUVER (NCT05804045), a double-blind, international, placebo-controlled phase 3 trial in which patients with symptomatic TGCT and at least 1 measurable lesion of 2 cm or more were randomly assigned 2:1 to pimicotinib 50 mg or placebo once daily.^1,2

According to the FDA’s news release, the objective response rate (ORR) at week 25 by blinded independent central review was approximately 56% (95% CI, 42-68) with pimicotinib versus 3.2% (95% CI, 0.1-17) with placebo. Median duration of response (DOR) was not reached; however, 32 responders (91%) had a DOR of at least 6 months, and 27 (77%) had a DOR of at least 12 months.¹

The primary analysis published in The Lancet, which included 94 patients (63 pimicotinib, 31 placebo) and had a data cutoff of September 23, 2024, reported an ORR of approximately 54% (34 of 63) versus 3% (1 of 31). The FDA did not explain the difference.^1,2

Patient-centered outcomes also improved significantly at week 25. In the published analysis, the least-squares mean difference versus placebo was about 15.7 points for relative range of motion (P = .0003), –2.1 points for Brief Pain Inventory (BPI) worst pain (P < .0001), and 3.4 points for Patient-Reported Outcomes Measurement Information System Physical Function score (P = .0074). A BPI-30 pain response—at least a 30% reduction in worst pain without increased analgesic use—occurred in 64% of patients receiving pimicotinib vs 16% receiving placebo.²

Safety and Pharmacist Considerations

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

In MANEUVER, CPK elevation was the only grade 3 or 4 AE occurring in more than 10% of patients receiving pimicotinib (13%). Dose reductions and discontinuations occurred in approximately 8% and 2% of these patients, respectively, and investigators reported no cholestatic hepatotoxicity, drug-induced liver injury, or skin or hair hypopigmentation.²

Labeling carries warnings for hepatotoxicity and embryo-fetal toxicity. Liver tests are required before starting treatment, monthly for the first 3 months, every 3 months for the rest of the first year, and as needed thereafter. Patients who can become pregnant need a pregnancy test before starting, and they and male patients with partners who can become pregnant should use effective contraception during treatment and for 2 weeks after the last dose. Breastfeeding is not recommended over the same period. The FDA also warns of increased serum creatinine without an effect on renal function. Pimicotinib is dosed at 50 mg orally once daily, with or without food.^1,4

Positioning Among CSF-1R Inhibitors

Pexidartinib (Turalio; Daiichi Sankyo) carries a risk of fatal cholestatic hepatotoxicity, requires enrollment in an FDA Risk Evaluation and Mitigation Strategy program, and is taken twice daily with dietary requirements, whereas vimseltinib (Romvimza; Deciphera Pharmaceuticals) is given twice weekly with at least 72 hours between doses. Week-25 ORRs were approximately 39% in the pivotal ENLIVEN trial of pexidartinib and 40% in the MOTION trial of vimseltinib; however, no head-to-head trials have compared pimicotinib with either agent.²

REFERENCES

1. FDA approves pimicotinib for symptomatic tenosynovial giant cell tumor. News release. FDA. November 10, 2026. Accessed November 11, 2026. https://www.fda.gov/drugs/cancer-approvals/fda-approves-pimicotinib-symptomatic-tenosynovial-giant-cell-tumor

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;407:1072-1083. doi:10.1016/S0140-6736(25)02602-9

3. Pimicotinib approved as systemic treatment in China for tenosynovial giant cell tumor. News release. Merck KGaA, Darmstadt, Germany. December 22, 2025. Accessed November 11, 2026. https://www.emdgroup.com/en/news/pimicotinib-china-approval-22-12-2025

4. FDA approves PIMJETMI (pimicotinib) for patients with tenosynovial giant cell tumor. News release. EMD Serono. November 10, 2026. Accessed November 11, 2026. https://www.emdserono.com/us-en/company/news/press-releases/fda-approves-pimjetmi-for-tgct-11-10-2026.html