ABBISKO-B(02256) Betjemai NDA accepted by FDA for the treatment of TGCT

ABBISKO-B (02256) announcement, on January 13, 2026, Shanghai Heyu Biomedical Technology Co., Ltd. (Heyu Medicine) announced that its independently developed novel, orally administered, highly selective and effective small molecule colony-stimulating factor 1 receptor (CSF-1R) inhibitor Bejimai (pamitinib hydrochloride capsule, ABSK021) for the systemic treatment of tenosynovial giant cell tumor (TGCT) patients' new drug application (NDA) has been formally accepted by the US Food and Drug Administration (FDA). Bejimai was independently developed by Heyu Medicine and authorized by Merck for its global commercialization. In December 2025, Bejimai was approved for listing by the China National Medical Products Administration (NMPA). It is used for symptomatic TGCT adult patients who may undergo surgery that could result in functional limitations or severe complications. Applications for listing in other markets are currently under review by the relevant regulatory agencies. The acceptance of Bejimai's NDA by the FDA was based on the positive effectiveness and safety results of the global multicenter, randomized, double-blind, placebo-controlled phase III MANEUVER study. The study showed that TGCT patients who received once-daily oral Bejimai achieved a statistically significant improvement in the primary endpoint, the objective response rate (ORR) assessed at week 25 based on the RECIST v1.1 criteria by the blinded independent review committee (BIRC). Furthermore, all secondary endpoints related to key clinical outcomes in TGCT patients showed significant and clinically meaningful improvements, including range of joint motion, physical function improvement, as well as reduced stiffness and pain. Long-term data with a median follow-up of 14.3 months further demonstrated sustained improvement in ORR for patients receiving Bejimai treatment since the start of the study. TGCT is a rare locally aggressive tumor that occurs in or around joints, leading to gradual swelling, stiffness, and limited mobility in the affected joints, significantly impacting patients' daily activities and quality of life. Without timely treatment or in case of recurrence, TGCT can cause irreversible damage to bones, joints, and surrounding tissues. With the continuous regulatory submissions of Bejimai in major markets worldwide, the drug is expected to provide a once-daily, oral, effective, and well-tolerated treatment option for TGCT patients globally, helping to meet the unmet clinical needs in this disease area.