Introduction
The FDA has granted approval to Ono Pharmaceutical’s Romvimza for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT), a typically noncancerous growth affecting the joints. This approval offers a new option for patients with TGCT in whom surgery would potentially worsen bodily function or cause severe illness.
Romvimza, also an inhibitor of the colony-stimulating factor 1 receptor (CSF1R), joins Daiichi Sankyo’s Turalio as an approved therapy for TGCT. Turalio was approved in 2019. Ono acquired Romvimza through the $2.4 billion purchase of Deciphera Pharmaceuticals. Ono’s CEO, Gyo Sagara, highlighted the acquisition as a means to broaden the company’s targeted oncology portfolio, expedite U.S. and European business development, and enhance kinase drug discovery capabilities
Efficacy and Safety
The approval of Romvimza is based on the MOTION trial. In the trial, 40% of TGCT patients who were not suitable for surgery responded to Romvimza at Week 25, compared to 0% in the placebo group. After six months of additional follow-up, 85% of responders maintained their response for at least six months, and 58% maintained it for at least nine months. The study also showed statistically significant improvements in active range of motion, patient-reported physical functioning, and patient-reported pain.
In 2019, Daiichi’s Turalio secured FDA approval with a 39% overall response rate at Week 25, versus 0% for placebo, also in TGCT patients for whom surgery was not recommended. Among patients followed for at least six months, 96% maintained their responses for at least six months.
Dosage and Administration
Romvimza offers a more convenient dosing schedule of 30 mg twice weekly, compared to Turalio’s 250 mg twice daily. Both medications are administered orally.
Safety Profile
Turalio carries a black box warning due to concerns about “serious and potentially fatal liver injury” and is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program. The data monitoring committee stopped enrollment early in Turalio’s phase 3 trial after noticing cholestatic liver toxicity. In the MOTION trial, no evidence of cholestatic hepatotoxicity or drug-induced liver injury was observed with Romvimza. Ten percent of patients receiving Romvimza experienced treatment-emergent grade 3 or 4 elevated blood creatine phosphokinase, indicating muscle injury or stress. Romvimza’s label doesn’t include any boxed warning or REMS requirement.
