FDA Fully Approves Deciphera's ROMVIMZA™ for TGCT Treatment

Ono Pharmaceutical Co., Ltd., headquartered in Osaka, Japan, and its subsidiary Deciphera Pharmaceuticals have announced the approval of their drug ROMVIMZA™ (vimseltinib) by the U.S. Food and Drug Administration (FDA). This approval marks a significant step forward in the treatment of tenosynovial giant cell tumor (TGCT), particularly for adult patients for whom surgical options pose risks of increased functional limitations or severe complications.

ROMVIMZA, a kinase inhibitor, was developed using Deciphera’s proprietary switch-control kinase inhibitor platform. The drug's approval followed its successful performance in clinical trials, including the pivotal Phase 3 MOTION study. This trial highlighted ROMVIMZA's efficacy and safety, demonstrating its ability to significantly shrink tumors and improve patients' quality of life while maintaining a well-tolerated safety profile. Notably, the study showed a substantial objective response rate (ORR) at Week 25 compared to the placebo, with significant enhancements in the active range of motion and physical functioning, and a reduction in patient-reported pain levels.

The success of ROMVIMZA’s clinical trials underscores its potential as a new standard of care for TGCT patients, providing an alternative to surgical intervention, which can often lead to severe morbidity. Hans Gelderblom, M.D., Ph.D., from Leiden University Medical Center, emphasized the drug’s potential to fulfill unmet needs within the TGCT community, noting its distinct ability to improve various quality-of-life measures without the liver injury risks associated with other treatments.

TGCT is a rare, non-cancerous tumor that develops in or around joints, caused by a genetic mutation that results in the overproduction of colony-stimulating factor 1 (CSF1). Although non-malignant, TGCT can cause significant damage to joints and surrounding tissues if left untreated, often leading to pain, swelling, and reduced mobility. Surgical resection has been the primary treatment but faces limitations due to recurrence and potential complications. ROMVIMZA represents a crucial advancement for patients who are not viable candidates for surgery.

The FDA's approval of ROMVIMZA also marks a significant milestone for Deciphera Pharmaceuticals, as it is the second therapy approved through its innovative switch-control kinase inhibitor platform. Ryota Udagawa, President and CEO of Deciphera, expressed gratitude to everyone involved in the drug's development, acknowledging the collaborative effort that led to this achievement. The company is committed to making ROMVIMZA available to patients in the U.S. shortly and is actively working on ensuring its accessibility.

In addition to its U.S. approval, ROMVIMZA is also under review by the European Medicines Agency (EMA) following a marketing authorization application submitted by Deciphera in July 2024. The company is dedicated to supporting TGCT patients and healthcare providers through its Deciphera AccessPoint™ program, which offers comprehensive access and financial assistance for eligible patients.

ROMVIMZA's approval is a promising development for TGCT treatment, offering a new therapeutic option for patients with limited surgical possibilities. This advancement not only addresses a significant unmet need but also exemplifies the potential of Deciphera's innovative approach to drug development, expanding treatment options for complex medical conditions.