FDA approves pimicotinib for adults with symptomatic tenosynovial giant cell tumor
The FDA approved pimicotinib (Pimjetmi) on November 10, 2026, after an overall response rate of 56% versus 3.2% on placebo at Week 25. For adults with symptomatic TGCT, an approved kinase inhibitor now exists.
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The Food and Drug Administration approved pimicotinib (Pimjetmi, EMD Serono, Inc.), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity, the FDA said. In the MANEUVER trial, the overall response rate at Week 25 was 56% in the pimicotinib arm and 3.2% in the placebo arm, according to the agency. The recommended dosage is 50 mg orally once daily, the FDA said.
| Arm | Part 1 participants | ORR at Week 25 (FDA) | ORR at Week 25 (registry) |
|---|---|---|---|
| Pimicotinib | 63 | 56% (95% CI: 42, 68) | 54.0% (95% CI 40.9–66.6) |
| Placebo | 31 | 3.2% (95% CI: 0.1, 17) | 3.2% (95% CI 0.1–16.7) |
From the FDA approval notice of November 10, 2026 and the trial registry record NCT05804045, results first posted 2026-05-04.
Why it matters
The FDA page states that full prescribing information for Pimjetmi will be posted on Drugs@FDA, so the label text is not yet available from that page. The approval covers adults whose tumor is symptomatic and for whom surgical resection may cause worsening functional limitation or severe morbidity. The FDA said pimicotinib received fast track and breakthrough therapy designation. The trial registry record lists the MANEUVER trial as a Phase 3 study sponsored by Abbisko Therapeutics Co, Ltd.
Overall response rate and duration of response
The FDA gave November 10, 2026 as the approval date. The agency lists these measures:
- Overall response rate (ORR) at Week 25, assessed by blinded independent central review (BICR) per RECIST v1.1: 56% (95% CI: 42, 68) in the pimicotinib arm and 3.2% (95% CI: 0.1, 17) in the placebo arm.
- Median duration of response (DOR): not reached in the pimicotinib arm.
- Number of responders with a DOR of at least 6 months: 32 responders (91%).
- Number of responders with a DOR of at least 12 months: 27 (77%).
The FDA also said statistically significant improvements were observed in the pimicotinib arm compared to the placebo arm at Week 25 across prespecified patient-reported and functional endpoints. Patients treated with pimicotinib experienced gains in active range of motion of the affected joint as measured by goniometry assessments, a reduction in worst pain on the Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS), and improved physical functioning on the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function T-score.
MANEUVER trial design and participants
The FDA said efficacy was evaluated in MANEUVER (NCT05804045), a double-blind, randomized (2:1), placebo-controlled, international trial in patients with TGCT for whom surgical resection may cause dysfunction or serious complications. Eligible patients had a confirmed diagnosis of symptomatic TGCT with measurable disease with at least one lesion having a minimum size of 2 cm. Patients were randomized to pimicotinib 50 mg or placebo once daily, and randomization was stratified by geographic region (China versus non-China).
The trial registry record lists an actual enrollment of 94 participants. Part 1 started with 63 participants in the pimicotinib arm and 31 in the placebo arm. Pimicotinib or matching placebo was given for 24 weeks in Part 1, which was blinded. Pimicotinib was then given open-label in Part 2 and Part 3, and the placebo arm had the option to receive open-label pimicotinib. The record lists masking as quadruple (participant, care provider, investigator, outcomes assessor).
The record's primary outcome is Objective Response Rate (ORR), assessed by central read using Response Evaluation Criteria in Solid Tumors (RECIST) (Version 1.1), from baseline to Week 25. For Part 1 the registry results list 54.0% (95% CI 40.9–66.6) with pimicotinib and 3.2% (95% CI 0.1–16.7) with placebo, a risk difference of 50.7 and p<0.0001 (Fisher exact). The record says results were first posted 2026-05-04. The registry figures differ slightly from the FDA figures above; each is reported as the source lists it.
Safety
The FDA said the most common (>20%) adverse reactions were pruritus, rash, face edema, periorbital edema, fatigue, nausea, headache, and peripheral edema. The most common (>30%) lab abnormalities were creatine phosphokinase increased, lactate dehydrogenase increased, aspartate aminotransferase increased, lipase increased, cholesterol increased, amylase increased, and alanine aminotransferase increased. The prescribing information includes warnings and precautions for hepatotoxicity, embryo-fetal toxicity, and increased serum creatinine without affecting renal function.
Project Orbis review
The FDA said the review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. For this review, the FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada (HC), Israel's Ministry of Health (ImoH), Switzerland's Swissmedic (SMC), and the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA). The European Medicines Agency (EMA) was an official observer. The review also used the Assessment Aid, a voluntary submission from the applicant to help the FDA with its assessment.
What remains uncertain
- The FDA page states that the median duration of response was not reached in the pimicotinib arm (FDA approval notice).
- The FDA page states that applications at the other regulatory agencies in the review are either in the submission process or under review (FDA approval notice).
- The trial registry record lists the study as active, not recruiting, with study completion listed as 2028-06 (estimated) (study record).