Vimseltinib

The asset is intended at treating tenosynovial giant cell tumours. Credit: Gorodenkoff via Shutterstock. The US Food and Drug Administration (FDA) has accepted for priority review the new drug application (NDA) for Ono Pharmaceutical subsidiary Deciphera Pharmaceuticals ’ vimseltinib, a treatment for tenosynovial giant cell tumours (TGCTs). Vimseltinib, designed to hinder the colony-stimulating factor 1 receptor (CSF1R), is a product of Deciphera’s switch-control kinase inhibitor platform. The US regulator set a Prescription Drug User Fee Act goal date of 17 February 2025. The FDA’s decision is underpinned by results from the Phase III MOTION study, a two-part, randomised, double-blind, placebo-controlled trial. It assessed vimseltinib’s efficacy and safety in patients with TGCT not amenable to surgery and without prior anti-CSF1/CSF1R therapy, except for imatinib or nilotinib. See Also: Pfizer and BioNTech’s Covid-19/flu combination vaccine falters in Phase III readout AstraZeneca wins approval for Imfinzi in NSCLC perioperative setting At week 25, the asset offered a statistically significant and clinically meaningful objective response rate in the intent-to-treat population versus placebo. It also showed significant improvements in all key secondary endpoints over placebo. Vimseltinib’s safety profile in the trial has been deemed manageable, with safety data from the MOTION study aligning with earlier Phase I/II clinical trial findings. The European Medicines Agency (EMA) initiated the centralised review process for vimseltinib, having accepted a Marketing Authorization Application in July this year. Deciphera Pharmaceuticals CEO and president Steve Hoerter said: “Building upon positive results from the MOTION pivotal Phase III study and following our recent announcement that EMA review of the vimseltinib MAA has begun, we are excited to initiate the regulatory review process in the US and we look forward to working with the FDA to deliver a new treatment option to patients with TGCT.” In April this year, Ono and Deciphera signed a definitive merger agreement for a deal valued at $2.4bn.

Vimseltinib is intended to treat tenosynovial giant cell tumour. Credit: crystal light / Shutterstock. The European Medicines Agency (EMA) has accepted for review the marketing authorisation application (MAA) of Ono Pharmaceutical subsidiary Deciphera Pharmaceuticals ‘ vimseltinib to treat tenosynovial giant cell tumour (TGCT). Vimseltinib is a colony-stimulating factor 1 receptor (CSF1R). The review will be carried out under the centralised review process of the European regulator for all 27 member states of the European Union (EU), Iceland, Norway and Liechtenstein. The MAA filing is based on the findings of the Phase III MOTION clinical trial assessing the safety and efficacy of vimseltinib in people with TGCT not amenable to surgical procedure without previous anti-CSF1/CSF1R therapy, versus placebo. The study showed a significant objective response rate (ORR) at week 25 – the trial’s primary endpoint – compared to placebo. See Also: Merck secures FDA authorisation for Ebola vaccine Ervebo US proposes rules for drug importation from Canada Secondary endpoints of the study included ORR per tumour volume score, active range of motion, physical function and quality of life assessments. Vimseltinib also showed a manageable safety profile with the safety findings from MOTION in line with data reported in the Phase I/II trial of vimseltinib. Developed using Deciphera’s switch-control kinase inhibitor platform, vimseltinib is an investigational, oral tyrosine kinase inhibitor designed to selectively inhibit CSF1R. It received orphan drug designation from the EMA in December 2019. Deciphera Pharmaceuticals president and CEO Steve Hoerter stated: “Building upon positive results from the MOTION pivotal Phase III study, we are excited to initiate the regulatory review process in the EU and we are one step closer in our mission to bring vimseltinib to TGCT patients in need of an effective and well-tolerated treatment.”