WHATNEXT partner website Patient Worthy, your leading source for rare disease news, reported that an investigational drug named pimicotinib has earned the European Medicines Agency (EMA) Orphan Drug designation as a treatment for tenosynovial giant cell tumors (TGCT).
Author Jessica Lynn notes:
So far, pimicotinib has performed positively in the clinical setting. Updated data from a Phase 1b study show that 50mg pimicotinib once daily led to an objective response rate of 87.5%, compared to 66.7% when taking 25mg daily. Objective response rate refers to how many people within a trial had a partial or complete response to treatment. In this case, there were three complete responses in the 50mg group and two in the 25mg group.
Check out the full article here.
Editor’s Note: Get Involved
Cancer doesn’t discriminate. Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a cancer journey to share, reach out here to learn more about how your voice can help spread awareness and inspire individuals from all walks of life.
EMA news Orphan Drug tenosynovial giant cell tumors TGCT treatment
Last modified: March 12, 2024
