FDA Approves Merck KGaA Tumour Pill After 56% Respond in Trial

The US Food and Drug Administration has approved pimicotinib, sold as Pimjetmi, for adults with symptomatic tenosynovial giant cell tumour (TGCT). EMD Serono, the US and Canadian healthcare business of Germany's Merck KGaA, announced the approval. The drug came from Chinese biotech Abbisko Therapeutics.

What happened

The label covers patients for whom surgery to remove the tumour could worsen function or cause severe complications. Pimicotinib is a pill that blocks a receptor called CSF-1R. The approved dose is 50 mg by mouth once a day, according to Targeted Oncology.

The approval rests on MANEUVER, a phase 3 trial run by Abbisko. It randomized 94 patients, with 63 on pimicotinib and 31 on placebo for 24 weeks. The company said 49 patients enrolled in the US, Canada and Europe, and 45 in China.

At week 25, 56% of patients on the drug had a tumour response, compared with 3% on placebo, the company said. Targeted Oncology, citing the trial's Lancet paper, put the figure at 54.0%, or 34 of 63 patients. In longer follow-up of the 63 patients first given the drug, with a median of 14.3 months, the response rate was 76.2%, Targeted Oncology reported.

The most common side effects included itching, rash, swelling of the face and around the eyes, fatigue, nausea and headache. The label carries warnings for liver injury and harm to a developing fetus. In the blinded part of the trial, one patient on the drug stopped because of side effects.

Why it matters

Pimicotinib joins pexidartinib and vimseltinib as FDA-approved drugs of the same type for TGCT, Targeted Oncology noted. That makes three approved options in the class.

The US is the drug's fourth market, RTTNews reported. China approved it in December 2025, Health Canada in June 2026 and Swiss regulators in October 2026, the company said. The FDA review ran under Project Orbis, the company said.

What it means for people in the industry

Merck KGaA holds worldwide sales rights. EMD Serono says it has about 1,050 employees, with commercial, clinical and research work in Massachusetts. The company did not announce new jobs with the approval.

For Abbisko, a milestone payment is due after the first US sale. It is also eligible for double-digit royalties on global sales, RTTNews reported. Watch for the US launch date and price, which were not in the announcements.

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Sources

• FDA Approves PIMJETMI (pimicotinib) for Patients with Tenosynovial Giant Cell Tumor, EMD Serono via Business Wire
• FDA Approves Pimicotinib for Symptomatic TGCT, OncLive
• FDA Approves Pimicotinib for Symptomatic Tenosynovial Giant Cell Tumor, Targeted Oncology
• FDA Approves Abbisko's PIMJETMI For Tenosynovial Giant Cell Tumor, RTTNews