The U.S. FDA has approved Ono Pharmaceutical’s kinase inhibitor, Romvimza, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.
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.
Surgery is the main treatment option for TGCT, however, for a subset of patients, surgical removal will potentially cause worsening functional limitation or severe morbidity. For this group of patients, systemic treatment options are limited. Romvimza oral capsules — developed by Ono’s subsidiary, Deciphera Pharmaceuticals — will provide a new therapeutic option.
Romvimza will compete with Daiichi Sankyo’s TGCT capsules, branded Turalio, which won FDA approval in 2019.
Japan-based Ono's 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, compared to placebo, as well as the phase 1/2 study of Romvimza. In the MOTION trial, Romvimza demonstrated a statistically significant and clinically meaningful overall response rate at week 25 in the intent-to-treat population.
The marketing authorization application for Romvimza for TGCT is currently under review by the EMA.