The US Food and Drug Administration (FDA) approved pimicotinib (Pimjetmi, EMD Serono, Inc.) on November 10, 2026, for adults with symptomatic tenosynovial giant cell tumor (TGCT) for whom surgical resection may worsen functional limitations or cause severe morbidity.
The approval was supported by findings from MANEUVER (NCT05804045), an international, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of pimicotinib in patients with TGCT for whom surgery could result in functional impairment or serious complications, according to the FDA.
Eligible participants had a confirmed diagnosis of symptomatic TGCT with measurable disease, including at least 1 lesion measuring 2 cm or larger. Patients were randomly assigned in a 2:1 ratio to receive pimicotinib 50 mg or placebo orally once daily. Randomization was stratified by geographic region, comparing patients enrolled in China with those enrolled outside China.
The FDA identified pruritus, rash, facial edema, periorbital edema, fatigue, nausea, headache, and peripheral edema as the most common adverse reactions, each occurring in more than 20% of patients. Laboratory abnormalities reported in more than 30% included elevated creatine phosphokinase, lactate dehydrogenase, aspartate aminotransferase, lipase, cholesterol, amylase, and alanine aminotransferase levels.
The prescribing information includes warnings and precautions for hepatotoxicity, embryo-fetal toxicity, and increased serum creatinine without an associated effect on renal function. The recommended dosage is 50 mg orally once daily, with or without food.
The FDA reviewed pimicotinib through Project Orbis, collaborating with regulatory authorities in Australia, Canada, Israel, Switzerland, and the United Kingdom, with the European Medicines Agency participating as an observer. The kinase inhibitor previously received fast-track and breakthrough therapy designations. The agency also used its Assessment Aid program to facilitate regulatory evaluation.
Source: FDA
