Beijing,Monday,June 17,2024 – In a meaningful win for patients battling a rare bone tumor,Chinese health authorities have approved Merck KGaA’s pimicotinib as a treatment for tenosynovial giant cell tumor (TGCT) in adults when surgery poses substantial risks.This approval marks a turning point, offering a new systemic therapy option for those facing functional limitations or severe health consequences from surgical intervention.
Pimicotinib Demonstrates Unprecedented Response Rates in clinical Trials
The approval follows a priority review and is based on phase 3 study results showing the highest objective response rate (ORR) ever observed in a TGCT systemic treatment trial.
After 25 weeks of treatment, pimicotinib achieved a statistically significant betterment in ORR – the primary endpoint assessed by a blinded independent committee (BIRC) – compared to placebo (54% versus 3.2%).these results suggest a substantial benefit for patients who previously had limited treatment options.
Did you know? TGCTs, while not cancerous, can be locally aggressive, meaning they grow and destroy nearby tissue. They account for approximately 3-5% of all primary bone tumors.
Beyond the primary endpoint, pimicotinib also demonstrated clinically meaningful and statistically robust improvements in secondary endpoints, notably in patients’ relative range of motion, impacting their daily lives. This suggests the treatment not only shrinks the tumor but also helps restore function.
A Novel Drug Developed by Abbisko Therapeutics
Pimicotinib, developed by Chinese biotech firm Abbisko Therapeutics, functions as an inhibitor of the colony-stimulating factor receptor type 1 (CSF-1R). It is the first class 1 chemical drug approved in China specifically for TGCT, signaling a new era in targeted therapies for this challenging condition.
What is TGCT? Tenosynovial giant cell tumor is a rare, locally aggressive bone tumor that typically affects young adults. It often occurs near joints and can cause pain, swelling, and limited movement.
Pro tip Clinical trials often measure “objective response rate” (ORR) to determine if a treatment is working. It refers to the percentage of patients whose tumors shrink or disappear.
The drug has also garnered significant international attention, receiving Breakthrough Therapy Designation (BTD) from the Food and Drug Administration (FDA) in the United States and Priority Medicine Designation (PRIME) from the European Medicines Agency (EMA), paving the way for potential approvals in those regions as well.
Reader question What impact do you think this approval will have on TGCT treatment globally? Share your thoughts!
- Pimicotinib is approved in China for TGCT patients for whom surgery is high-risk.
- The phase 3 trial showed a 54% ORR with pimicotinib versus 3.2% with placebo.
- The drug is developed by Abbisko therapeutics and targets CSF-1R.
