Romvimza (vimseltinib) / Ono Pharmaceutical - LARVOL DELTA

Romvimza: “The Committee adopted a positive opinion recommending the granting of a marketing authorisation by consensus together with the CHMP assessment report and translation timetable”; Tenosynovial giant cell tumour (European Medicines Agency) - CHMP Final Minutes of the meeting on 21 - 24 Jul 2025: “The legal status was agreed as medicinal product subject to restricted medical prescription”

CHMP • Oncology • Tenosynovial Giant Cell Tumor

Evaluate the Effect of Vimseltinib on Organic Cation Transporter 2 (OCT2) (clinicaltrials.gov) - P1 | N=22 | Completed | Sponsor: Deciphera Pharmaceuticals, LLC | Active, not recruiting ➔ Completed | Trial completion date: Jun 2026 ➔ Jan 2026 | Trial primary completion date: Jun 2026 ➔ Jan 2026

Trial completion • Trial completion date • Trial primary completion date

Long-term efficacy and safety of vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT): 2-year results from the MOTION phase 3 trial (DKK 2026) - No abstract available

Clinical • P3 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

Long-term clinical outcome assessments (COAs) in patients (pts) with tenosynovial giant cell tumor (TGCT) treated with vimseltinib: 1-year results from the MOTION phase 3 trial (DKK 2026) - No abstract available

Clinical • Clinical data • P3 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

A Phase 2, Open Label Study to Evaluate Vimseltinib in Adults with Active Chronic Graft-Versus-Host Disease after Failure of Prior Systemic Therapies (TCT-ASTCT-CIBMTR 2026) - P2 | "Patients must have failed ≥2 prior lines of systemic therapy and can only be on systemic corticosteroid use ≤1 mg/kg/day prednisone equivalent and 1 additional immunosuppressive agent, provided doses are stable for ≥2 weeks prior to starting vimseltinib. To describe an ongoing Phase 2 study evaluating vimseltinib therapy in adults with active moderate-to- severe cGVHD. Understand type of study: This is a Phase 2, open-label, dose-finding, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of biweekly vimseltinib in adults with active cGVHD after prior systemic therapy failure."

Clinical • P2 data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Fibrosis • Gastrointestinal Disorder • Giant Cell Tumor of Bone • Graft versus Host Disease • Immunology • Infectious Disease • Solid Tumor • Tenosynovial Giant Cell Tumor

Mixed-Methods to Define Meaningful Change using Exit Interviewand Clinical Trial Data in Patients with Tenosynovial Giant Cell Tumor (TGCT). (PubMed, Qual Life Res) - P3 | "The responder definitions were at least a 3-point improvement for PROMIS-PF, a 10% improvement for active ROM, and a 2-point improvement for the Worst Stiffness NRS. Qualitative interviews facilitate integrating the patient perspective in the selection of anchors and defining meaningful change."

Clinical • Interview • Journal • Giant Cell Tumor of Bone • Oncology • Pain • Tenosynovial Giant Cell Tumor

Properties of FDA-approved small molecule protein kinase inhibitors: a 2026 update. (PubMed, Pharmacol Res) - "The following ten drugs received FDA approval in 2025 - avutometinib (inhibiting MEK1/2 in serous ovarian carcinomas), defactinib (blocking FAK in low grade serous ovarian carcinomas), delgocitinib (antagonizing the JAK family in hand eczema), mirdametinib (inhibiting MEK1/2 in type I neurofibromatosis), remibrutinib (blocking BTK in chronic spontaneous urticaria), rilzabrutinib (antagonizing BTK in chronic immune thrombocytopenia), sunvozertinib (blocking mutant exon 21 insertion EGFR NSCLC), taletrectinib (inhibiting mutant ROS1 in NSCLC), vimseltinib (blocking CSF1R in tenosynovial giant cell tumors), and zongertinib (antagonizing mutant HER2 in NSCLC). This article summarizes the physicochemical properties of all 94 FDA-approved small molecule protein kinase inhibitors including the molecular weight, number of hydrogen bond donors/acceptors, ligand efficiency, lipophilic efficiency, polar surface area, and solubility. A total of 45 of the 94 FDA-approved drugs have a..."

FDA event • Journal • Review • Atopic Dermatitis • Chronic Spontaneous Urticaria • Contact Dermatitis • Dermatology • Genetic Disorders • Giant Cell Tumor of Bone • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Lung Cancer • Neurofibromatosis • Non Small Cell Lung Cancer • Oncology • Ovarian Cancer • Ovarian Serous Adenocarcinoma • Solid Tumor • Tenosynovial Giant Cell Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Urticaria • CSF1R • EGFR • HER-2 • ROS1

Management of tenosynovial giant cell tumor: approved and investigational therapies. (PubMed, Expert Rev Anticancer Ther) - "Pexidartinib and vimseltinib demonstrate comparable efficacy in TGCT with objective response rates (ORR) of 39% and 40%, respectively, at week 25 of treatment; however, vimseltinib offers improved hepatic safety and tolerability, supporting its use as the preferred first-line systemic therapy. Emerging agents, including pimicotinib and emactuzumab, show potential for higher response rates and favorable safety profiles and may further reshape the TGCT treatment paradigm pending phase 3 trial results."

Journal • Review • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

A phase I study of the CSF1R inhibitor vimseltinib in combination with the PD-L1 inhibitor avelumab in patients with advanced sarcoma. (PubMed, ESMO Open) - "Vimseltinib plus avelumab was generally safe and well tolerated. This combination had minimal clinical efficacy in our population of heavily pretreated patients with sarcoma."

Journal • P1 data • Oncology • Sarcoma • Solid Tumor

Recent FDA-approved kinase inhibitors for cancer therapy in 2025: A comprehensive review and perspectives. (PubMed, EXCLI J) - "The present review paper aims to provide a summary and a critical synthesis of the development, therapeutic potential, and clinical performance of novel of kinase inhibitors in oncology (i.e. zongeritinib, sunvozertinib, vimseltinib, mirdametinib, avutometinib and defactinib), authorized by the US Food and Drug Administration (FDA) in 2025, aiming to highlight both their transformative role and their inherent limitations. See also the graphical abstract(Fig. 1)."

FDA event • Journal • Review • Hematological Disorders • Hematological Malignancies • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

Investigating vimseltinib in tenosynovial giant cell tumors. (PubMed, Expert Opin Pharmacother) - "Furthermore, there is an absence of severe toxicities that have arisen with other agents in the TGCT treatment space, specifically liver failure. Vimseltinib is a favorable option for patients with TGCTs and further efforts to determine its place in the sequence of overall management are needed."

Journal • Review • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Tenosynovial Giant Cell Tumor • CSF1R

Efficacy, safety, and patient-reported outcomes of vimseltinib in patients with tenosynovial giant cell tumor: Results from the phase 3 MOTION trial. (ASCO 2024) - P3 | "Patients treated with vimseltinib experienced statistically significant and clinically meaningful improvements in ORR by RECIST v1.1 and by TVS, active ROM, physical function, stiffness, health status, and pain vs pbo. Vimseltinib is an effective, well-tolerated CSF1R-targeted therapy that demonstrates significant clinical benefit and improves functional health and QoL in pts with symptomatic TGCT not amenable to surgery."

Clinical • P3 data • Patient reported outcomes • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Pain • Tenosynovial Giant Cell Tumor • CSF1R

CSF1R Inhibition in Patients With Advanced Solid Tumors or Tenosynovial Giant Cell Tumor: A Phase 1 Study of Vimseltinib. (PubMed, Clin Cancer Res) - P1/2 | "Vimseltinib demonstrated long-term tolerability, manageable safety, dose-dependent exposure, and robust antitumor activity in patients with TGCT whose disease is not amenable to surgery."

Journal • Metastases • P1 data • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor • CSF1R

Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. (PubMed, Lancet) - P3 | "Vimseltinib produced a significant objective response rate and clinically meaningful functional and symptomatic improvement in patients with TGCT, providing an effective treatment option for these patients."

Journal • P3 data • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor

Updated efficacy and safety of vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT): One-year follow-up from the MOTION phase III trial (ESMO 2024) - P3 | "In this first report of long-term results (≥1 year) from MOTION, vimseltinib continued to be well tolerated and provided long-term antitumor responses in pts with TGCT not amenable to surgery."

Clinical • Late-breaking abstract • P3 data • Giant Cell Tumor of Bone • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor

Evaluate the Effect of Vimseltinib on the Pharmacokinetics of a BCRP and OATP1B1 Substrate (clinicaltrials.gov) - P1 | N=20 | Completed | Sponsor: Deciphera Pharmaceuticals, LLC | Active, not recruiting ➔ Completed | Trial completion date: Jun 2026 ➔ Dec 2025 | Trial primary completion date: Jun 2026 ➔ Dec 2025

Trial completion • Trial completion date • Trial primary completion date

Evaluate the Effect of Vimseltinib on the Pharmacokinetics of a BCRP and OATP1B1 Substrate (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: Deciphera Pharmaceuticals, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed

Evaluate the Effect of Vimseltinib on Organic Cation Transporter 2 (OCT2) (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: Deciphera Pharmaceuticals, LLC | Recruiting ➔ Active, not recruiting

Enrollment closed

EFFICACY WITH VIMSELTINIB IN PATIENTS (PTS) WITH TENOSYNOVIAL GIANT CELL TUMOR (TGCT) AND PRIOR COLONY-STIMULATING FACTOR 1 (CSF1) INHIBITOR THERAPY: A PHASE 2 CASE SERIES (CTOS 2025) - P1/2 | "Here, we report efficacy with vimseltinib in pts with prior pexidartinib or vimseltinib treatment. Pts with symptomatic TGCT not amenable to surgery were enrolled in this multicenter, open-label, phase 1/2 trial. These results suggest that pts with TGCT not amenable to surgery can derive clinical benefit from resumption of anti-CSF1R therapy, even after discontinuing the same (vimseltinib) or a different inhibitor. Vimseltinib retreatment was well tolerated with a manageable safety profile, consistent with prior reports."

P2 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1

EFFECT OF A HIGH-FAT MEAL ON THE PHARMACOKINETICS (PK) OF VIMSELTINIB, AN ORAL INHIBITOR OF THE COLONY-STIMULATING FACTOR 1 RECEPTOR (CSF1R), IN HEALTHY PARTICIPANTS (CTOS 2025) - P3 | "The 90% CIs for the GMRs of vimseltinib AUC0–tlast and AUC0–inf fell within the equivalence limits of 80% to 125%, indicating that administration of vimseltinib with a high-fat meal did not affect vimseltinib PK. Vimseltinib was well tolerated, and safety was consistent with prior reports. These results provide the basis for the approved labeling instruction that vimseltinib may be taken with or without food."

PK/PD data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

MEDICAL MANAGEMENT OF TENOSYNOVIAL GIANT CELL TUMOR (CTOS 2025) - "Multiple colony-stimulating factor 1 receptor inhibitors (CSF1Ris) have emerged for symptomatic pts, with pexidartinib (pexi) and vimseltinib (vim) approved. Here, we review phase 3 reports for pexi, vim, and pimicotinib (pimi). PubMed was searched for publications associated with TGCT, CSF-1Ri, therapies for treatment of TGCT, and known guidelines, with a focus on phase 3 trials and postmarket analyses... CSF1Ris show efficacy in TGCT. Pts should be monitored for potential side effects that are different for each presented drug. Further research to understand long-term side effects and duration of response will be paramount to optimize therapeutic strategies for pts with TGCT."

Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

LONG-TERM EFFICACY AND SAFETY OF VIMSELTINIB IN PATIENTS (PTS) WITH TENOSYNOVIAL GIANT CELL TUMOR (TGCT): RESULTS FROM THE MOTION PHASE 3 TRIAL (CTOS 2025) - No abstract available

Clinical • P3 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

EFFECT OF ITRACONAZOLE (ITZ) AND RABEPRAZOLE (RBP) ON THE PHARMACOKINETICS (PK) OF VIMSELTINIB, AN ORAL INHIBITOR OF THE COLONY-STIMULATING FACTOR 1 RECEPTOR, IN HEALTHY PARTICIPANTS (CTOS 2025) - "P-gp inhibition with ITZ and gastric acid suppression with RBP had weak effects on total vimseltinib exposure and absorption, respectively. Neither treatment affected the safety profile. Overall, the DDI for vimseltinib with ITZ or with RBP was not considered clinically significant, providing basis for approved concomitant use of vimseltinib with P-gp inhibitors and PPIs.Plasma PK Parameters of Vimseltinib by Treatment"

PK/PD data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

Deciphera Announces Multiple Data Presentations at the Connective Tissue Oncology Society (CTOS) Annual Meeting 2025 (Businesswire) - "Multiple presentations on vimseltinib for the treatment of Tenosynovial Giant Cell Tumor (TGCT), including encore data from the Phase 3 MOTION study presented in an oral session; Two posters on DCC-3009 in Gastrointestinal Stromal Tumor (GIST)."

Clinical data • Gastrointestinal Stromal Tumor • Tenosynovial Giant Cell Tumor

Health-Related Quality of Life Measured by EQ-5D Utility Among Patients With TGCT: A Trial-Based Analysis of EQ-5D Data From Patients in the MOTION Trial (ISPOR-EU 2025) - P3 | "TGCT patients not amenable for surgery in the MOTION trial had poor HRQoL at baseline (0.53) comparable to other pain-ridden diseases, such as osteoarthritis or multiple sclerosis. Based on MOTION, vimseltinib significantly improves PF and worst pain NRS over time, leading to large gains in HRQoL. These results fill a gap in describing HRQoL for TGCT patients not amenable for surgery and will support modeling the changes expected in EQ-5D due to changes in key symptoms of TGCT for health technology assessment."

Clinical • HEOR • CNS Disorders • Giant Cell Tumor of Bone • Immunology • Multiple Sclerosis • Osteoarthritis • Rheumatology • Tenosynovial Giant Cell Tumor