FDA Accepts Priority Review of Deciphera and Ono Pharmaceutical’s NDA for Vimseltinib to Treat TGCT
Friday, August 16, 2024
Ono Pharmaceutical has announced that the U.S. Food and Drug Administration (FDA) has accepted a priority review for the New Drug Application (NDA) for vimseltinib, a colony-stimulating factor 1 receptor (CSF1R) inhibitor.
Vimseltinib is an investigational oral tyrosine kinase inhibitor designed to selectively and effectively inhibit CSF1R. It was developed using Deciphera’s proprietary switch-control kinase inhibitor platform and is intended as a targeted treatment option for TGCT.
Vimseltinib, which is being developed by Deciphera Pharmaceuticals, Inc., a wholly-owned subsidiary of Ono, is intended for the treatment of patients with tenosynovial giant cell tumour (TGCT). The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of 17 February 2025 for the review. In mid-July, the European Medicines Agency (EMA) also accepted a Marketing Authorization Application (MAA) for vimseltinib and has begun its centralized review process.
In Part 1 of the study, patients were randomized to receive either vimseltinib or placebo for 24 weeks. In Part 2, patients initially assigned to the placebo group were given the option to receive vimseltinib, and all patients continued to receive vimseltinib in an open-label setting over a long-term period.
The NDA submission is based on data from the Phase 3 MOTION study, which assessed the efficacy and safety of vimseltinib in TGCT patients who are not candidates for surgery and have not received prior anti-CSF1/CSF1R therapy (although previous treatment with imatinib or nilotinib was allowed).
The study showed that vimseltinib achieved a statistically significant and clinically meaningful objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as evaluated by Blinded Independent Radiologic Review (BIRR) using Response Evaluation Criteria in Solid Tumours version 1.1 (RECIST v1.1), compared to placebo (40% in the vimseltinib group vs 0% in the placebo group, p <0.0001). Vimseltinib also showed significant improvements in all key secondary endpoints compared to placebo. The safety profile of vimseltinib was manageable and consistent with previous Phase 1/2 clinical trial data. The results of the MOTION study were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet.
The MOTION study is a two-part, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of vimseltinib in patients with TGCT who are not eligible for surgery and have not received prior anti-CSF1/CSF1R therapy. The study’s primary endpoint was the objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as assessed by Blinded Independent Radiologic Review (BIRR) using RECIST v1.1, compared to placebo. Secondary endpoints included tumour volume score (TVS), range of motion (ROM), physical function, stiffness, quality of life, and pain, all assessed at Week 25.
TGCT is a rare, non-malignant tumour that develops within or around joints, caused by a genetic translocation in the colony-stimulating factor 1 (CSF1) gene. This leads to the overexpression of CSF1 and the recruitment of CSF1R-positive inflammatory cells to the tumour site. Although benign, TGCT can cause significant pain, swelling, and restricted movement, and often recurs after surgery. For patients who are not candidates for surgery, systemic treatment options are limited, highlighting the need for new therapeutic approaches.
Source:businesswire.com
