FDA Grants Full Approval of Deciphera’s ROMVIMZA for TGCT Treatment
Ono Pharmaceutical Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has approved ROMVIMZA (vimseltinib), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity. The FDA previously granted Fast Track designation and Priority Review for ROMVIMZA, which was developed by Deciphera Pharmaceuticals, Inc. (“Deciphera”), a wholly owned subsidiary of Ono.
“The approval of ROMVIMZA provides a new, much-needed, well-tolerated, and effective treatment option for people suffering from TGCT,” said Hans Gelderblom, M.D., Ph.D., Chair of the Department of Medical Oncology at Leiden University Medical Center. “TGCT adversely affects the lives of patients, causing significant pain, limited mobility, and stiffness. The MOTION Phase 3 study demonstrated ROMVIMZA’s ability to shrink tumors along with being the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment. ROMVIMZA is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.”
“The FDA approval of ROMVIMZA for TGCT is a crucial advancement for the TGCT community and we believe ROMVIMZA has the potential to become the new standard of care for people with TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity. This is also an important milestone for our organization, as it is the second approved therapy discovered using Deciphera’s proprietary switch-control kinase inhibitor platform,” said Ryota Udagawa, President and Chief Executive Officer of Deciphera Pharmaceuticals. “I’d like to extend my gratitude to the patients, families, caregivers, and healthcare providers who contributed to the success in ROMVIMZA’s clinical studies. Their commitment, along with the dedication of the Deciphera and Ono teams, enabled us to advance this impactful new treatment, which we look forward to delivering to patients.”
TGCT is a rare, non-malignant tumor that forms within or near joints. TGCT arises from the dysregulation of the CSF1 gene, resulting in an overproduction of CSF1. If left untreated or if the tumor repeatedly recurs, it can lead to damage and degeneration in the affected joint and surrounding tissues, potentially causing significant disability.
The FDA approval was based on the efficacy and safety results from the pivotal Phase 3 MOTION study of ROMVIMZA in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of ROMVIMZA. In MOTION, ROMVIMZA demonstrated a statistically significant and clinically meaningful ORR at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (BIRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo (40% in ROMVIMZA arm vs 0% in placebo arm, p <0.0001). The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 25. The safety profile of ROMVIMZA is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.
