Merck’s Pimicotinib NDA Accepted by FDA for Rare Tumor Treatment

FDA Accepts Application for Systemic Treatment of TGCT

Merck has announced that the U.S. Food and Drug Administration (FDA) has accepted its new drug application (NDA) for pimicotinib as a systemic therapy for tenosynovial giant cell tumor (TGCT). This milestone marks an important step in the regulatory process, potentially bringing a new oral treatment option to patients with this rare and debilitating joint disease.

Based on Strong Clinical Trial Results

The FDA’s acceptance is supported by data from the global Phase III MANEUVER study, which demonstrated deep and durable tumor responses in people with TGCT treated with pimicotinib. Patients showed meaningful improvements in tumor size, symptom relief, and clinical outcomes over longer-term follow-up, underscoring the potential benefit of this therapy in cases where surgical options are limited.

Addressing Unmet Needs in TGCT

TGCT is a locally aggressive tumor that causes pain, stiffness, and reduced mobility around affected joints, significantly impacting quality of life. Current treatment options beyond surgery are limited, and recurrence after surgical resection is common. A successful NDA review could offer TGCT patients an effective, once-daily oral therapy that targets disease progression and symptom burden.

Global Development and Regulatory Activity

Pimicotinib was previously approved in China for symptomatic TGCT based on positive outcomes from the same MANEUVER study. With the FDA’s acceptance of the NDA, Merck now advances the regulatory path in the U.S., reflecting ongoing efforts to make the medicine available more widely across global markets.

Looking Ahead

Merck’s next steps will involve detailed review by the FDA, which will assess the full body of efficacy and safety evidence submitted by the company. If approved, pimicotinib could represent a meaningful new therapeutic option in the TGCT treatment landscape, offering patients an alternative to invasive procedures and prolonged recovery.