FDA approves Deciphera’s Romvimza for rare tumour type

Tenosynovial giant cell tumours can cause pain, stiffness, swelling and movement limitations

The US Food and Drug Administration (FDA) has approved Deciphera Pharmaceuticals’ Romvimza (vimseltinib) to treat adults with symptomatic tenosynovial giant cell tumours (TGCTs).

The drug has been specifically authorised for cases where surgical resection may potentially lead to worsening functional limitations or severe morbidity.

TGCTs are a group of rare, benign and typically non-life-threatening tumours that originate in the synovial lining of joints, bursae and tendon sheaths.

Patients can experience pain, stiffness, swelling and movement limitations, and the tumours can cause irreversible damage to surrounding tissues and structures of the affected limbs.

Romvimza is a tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R, which plays a key role in tumour growth.

The FDA’s decision on the drug was supported by positive results from the late-stage MOTION trial, which randomised 123 patients to receive a 30mg oral twice-weekly dose of Romvimza or placebo for 24 weeks. Patients could then continue with Romvimza treatment or switch from placebo to Romvimza during the study’s open-label period.

Overall response rate at week 25 was 40% in the Romvimza cohort and 0% in the placebo arm. Median duration of response (DOR) was not reached in the Romvimza group and, based on an additional six months of follow-up, 85% of responders had a DOR of at least six months and 58% had a DOR of at least nine months.

Significant benefits in active range of motion, and patient-reported physical functioning and pain were also seen in Romvimza-treated patients compared to those receiving placebo at week 25.