The European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for the tenosynovial giant cell tumors (TGCT) treatment vimseltinib, developed by Deciphera Pharmaceuticals, Inc., a wholly-owned subsidiary of Ono Pharmaceutical Co., Ltd. This significant milestone marks the beginning of the EMA’s centralized review process for vimseltinib, a colony-stimulating factor 1 receptor (CSF1R) inhibitor, for the treatment of patients with tenosynovial giant cell tumors. The review will encompass all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. Vimseltinib was previously granted Orphan Drug Designation by the EMA for the treatment of TGCT in December 2019.
The submission of the MAA is based on the positive results from the MOTION Phase 3 study. This pivotal study demonstrated that vimseltinib significantly improved the objective response rate (ORR) compared to placebo, with a statistically significant and clinically meaningful ORR at Week 25. The study included patients with tenosynovial giant cell tumors not amenable to surgery and who had not received prior anti-CSF1/CSF1R therapy. Vimseltinib showed a 40% ORR in the intent-to-treat population versus 0% for the placebo group, as assessed by Blinded Independent Radiologic Review (BIRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1). The safety profile of vimseltinib was manageable and consistent with previous clinical trial data.
Steve Hoerter, President and CEO of Deciphera Pharmaceuticals, expressed enthusiasm about the regulatory review process in the EU. “Building upon positive results from the MOTION pivotal Phase 3 study, we are excited to initiate the regulatory review process in the EU and are one step closer in our mission to bring vimseltinib to tenosynovial giant cell tumors patients in need of an effective and well-tolerated treatment,” Hoerter said.
MOTION Study Assesses Vimseltinib for Treating Tenosynovial Giant Cell Tumors Not Amenable to Surgery
The MOTION study is a comprehensive two-part, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to assess the efficacy and safety of vimseltinib in TGCT patients not amenable to surgery. The primary endpoint of the study was the ORR at Week 25, assessed by BIRR using RECIST v1.1 criteria. Secondary endpoints included ORR per tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain, all assessed at Week 25. In Part 1, patients were randomized to receive either vimseltinib or placebo for 24 weeks. In Part 2, patients initially on placebo could receive vimseltinib in an open-label setting for a long-term period.
Tenosynovial giant cell tumor is a rare, non-malignant tumor that develops inside or near joints. TGCT is caused by a translocation in the CSF1 gene, leading to overproduction of colony-stimulating factor 1 (CSF1) and recruitment of CSF1R-positive inflammatory cells into the lesion. Although benign, TGCT can grow and damage surrounding tissues, causing pain, swelling, and limited joint movement. Surgery is the primary treatment, but tumors often recur, particularly in diffuse-type tenosynovial giant cell tumors. For patients who are not candidates for surgery, systemic treatment options are limited, highlighting the need for new therapeutic interventions like vimseltinib.
Vimseltinib is an investigational, oral switch-control tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R. Developed using Deciphera’s proprietary switch-control kinase inhibitor platform, vimseltinib offers a novel approach to targeting the molecular drivers of tenosynovial giant cell tumors. The drug aims to provide a systemic treatment option for patients who have limited choices beyond surgery.
Deciphera Pharmaceuticals Advances Cancer Treatments with EMA Review of Vimseltinib for Tenosynovial Giant Cell Tumors
Deciphera Pharmaceuticals, now a subsidiary of Ono Pharmaceutical as of June 11, 2024, is dedicated to discovering, developing, and commercializing innovative medicines to improve the lives of people with cancer. The company leverages its expertise in kinase biology and its proprietary switch-control kinase inhibitor platform to develop a robust portfolio of treatments. Deciphera’s lead product, QINLOCK®, is already approved for the treatment of fourth-line gastrointestinal stromal tumor (GIST) and is available in numerous countries, including the EU and the US.
The acceptance of the MAA by the EMA initiates the centralized review process, which will cover all EU member states as well as Iceland, Liechtenstein, and Norway. This centralized approach is designed to streamline the approval process and ensure that patients across Europe have timely access to new, innovative treatments like vimseltinib. The acceptance of the MAA for vimseltinib marks a significant step forward in providing a new treatment option for patients with tenosynovial giant cell tumors. The ongoing review by the EMA will focus on the clinical efficacy and safety data provided by the MOTION study, to determine the drug’s suitability for widespread clinical use.
Vimseltinib Could Revolutionize Treatment for Tenosynovial Giant Cell Tumors Patients
If approved, vimseltinib could offer a critical new option for TGCT patients, particularly those who are not candidates for surgery. The ability to manage tenosynovial giant cell tumors with an oral medication that targets the underlying molecular mechanisms of the disease represents a major advancement in patient care. By potentially reducing tumor size and alleviating symptoms without the need for invasive procedures, vimseltinib could significantly improve the quality of life for many patients.
Deciphera and Ono remain committed to advancing their pipeline of innovative therapies and continuing their research into kinase inhibitors. The development of vimseltinib and its progression through clinical trials underscores the companies’ dedication to bringing forward new treatments that address unmet medical needs in oncology and other fields.
The European Medicines Agency’s acceptance of Deciphera’s MAA for vimseltinib is a crucial development in the fight against tenosynovial giant cell tumors. Supported by robust clinical trial data, vimseltinib has the potential to become a groundbreaking treatment for patients with this challenging condition. The ongoing review process will be closely watched by the medical community and patients alike, with the hope that vimseltinib will soon be available as a new standard of care for TGCT in Europe.
Resource: Deciphera, July 18, 2024
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