The United States Food and Drug Administration (FDA), which has responsibility for protecting and promoting US public health, has approved Vorasidenib as a first-in-class option for patients with IDH-mutant gliomas. A first-in-class medication is a prototype drug that uses a “new and unique mechanism of action” to treat a particular medical condition.
This approval for the treatment of IDH-mutant diffuse glioma is based on findings from the phase 3 INDIGO trial.
Treatment with Vorasidenib was found to significantly improve progression-free survival (PFS) and delayed the time to the next intervention (TTNI).
The approval is for the treatment of patients 12 and older with IDH-mutant, grade 2 astrocytoma or oligodendroglioma and marks the first approval of a systemic therapy for this patient cohort.
“What's exciting is this is a development that we haven't seen in over 20 years, and what we found is that when we looked at our patients that received Vorasidenib in comparison with the placebo, those patients had a much improved prognosis, such that their progression-free survival was extended to 27.7 months compared with 11 months, which is a very large, statistically significant difference,” explained Katherine B. Peters, MD, PhD, neuro-oncologist at Duke Cancer Center.
Commenting on this, our Director of Research, Policy and Innovation Dr Karen Noble said: “We are following the journey of Vorasidenib through the UK regulatory process with keen interest. We must have the UK at the forefront of market access to new therapeutics for brain tumour patients – for that not to be the case leaves patients and their families confused and anxious and on occasion taking choices to seek and self fund treatment overseas. That is a situation we cannot allow to continue.”
Read more on this story in Targeted Oncology.
Related reading:
- Access to promising drug for low-grade glioma a “priority”
- Clinical trial brings hope of a new treatment for low-grade glioma
Published Wednesday 7th August 2024.