On November 10, 2026, 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.

Full prescribing information for Pimjetmi will be posted on Drugs@FDA.

Efficacy and Safety

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. Randomization was stratified by geographic region (China versus non-China).

The major efficacy outcome measure was overall response rate (ORR), as assessed by blinded independent central review (BICR) per RECIST v1.1 at Week 25. ORR was 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) was not reached in the pimicotinib arm; 32 responders (91%) had a DOR ≥6 months and 27 (77%) had a DOR ≥12 months. In addition, 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 as measured by the Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS), and improved physical functioning as measured by the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function T-score.

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.

Recommended Dosage

The recommended pimicotinib dosage is 50 mg orally once daily with or without food.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada (HC), Israel’s Ministry of Health (ImoH), Switzerland’s Swissmedic (SMC), and United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The European Medicines Agency (EMA) was an official observer of this review. The applications may be undergoing the submission process or still be under review at the other regulatory agencies.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

Pimicotinib received fast track and breakthrough therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Source: FDA