The FDA has approved PIMJETMI (pimicotinib), a small-molecule colony-stimulating factor 1 receptor inhibitor, as a systemic treatment for adults with symptomatic tenosynovial giant cell tumor for which surgical resection may cause worsening functional limitation or severe morbidity. The drug was developed by Abbisko Therapeutics, which granted global commercialization rights to Merck KGaA, Darmstadt, Germany.
The approval is based on the global Phase 3 MANEUVER study and its open-label extension. PIMJETMI demonstrated a statistically significant improvement in objective response rate at week 25 as assessed by blinded independent review committee based on RECIST v1.1, with 56% versus 3% for placebo (p<0.0001). It also showed clinically meaningful and statistically significant improvements in relative range of motion (p=0.0003), physical functioning (p=0.0074) and pain (p<0.0001).
In longer-term follow-up with a median of 14.3 months for patients initially randomized to pimicotinib, presented at the 2025 ESMO Congress, the median duration of response was not reached. Seventy-seven percent of patients with response at week 25 maintained clinical benefit after an additional 12 months of follow-up.
The U.S. approval is the fourth for PIMJETMI, following approvals by China's NMPA in December 2025, Health Canada in June 2026 and Swissmedic in October 2026. The U.S., Canada and Switzerland applications were reviewed under Project Orbis.
Abbisko will receive a milestone payment upon first U.S. commercial sale, plus double-digit royalties on global net sales and potential regulatory, development and commercial sales milestone payments. TGCT is a rare, locally aggressive tumor that primarily affects the joints, tendon sheaths and bursae, causing pain, swelling, stiffness and limited joint mobility.
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