The FDA has approved vorasidenib, marketed as Voranigo by Servier Pharmaceuticals, for the treatment of Grade 2 astrocytoma or oligodendroglioma in patients with susceptible IDH1 or IDH2 mutations. This approval marks a significant milestone as the first systemic therapy specifically for this patient population.
The development of vorasidenib stems from a discovery made in 2008 by Dr. Darrell Bigner and a team at Johns Hopkins University, who identified a point mutation in the isocitrate dehydrogenase (IDH) gene within brain tumor tissues. This discovery led to a license that enabled the creation of vorasidenib, a drug that specifically targets the mutant IDH protein.
Clinical Trial Success
Clinical trials, including those conducted at Duke University, demonstrated the efficacy of vorasidenib. Dr. Katy Peters, a Professor of Neurology and Neurosurgery at Duke, highlighted that patients in the clinical trial experienced extended periods without any evidence of tumor progression. The trials also measured the time to next intervention (radiation, chemotherapy, or surgery), which was significantly delayed in patients receiving vorasidenib.
Rebecca Richmond, a trial participant diagnosed in 2016, shared her positive experience, noting the stability of her MRIs over four years. She also emphasized the benefit of an oral pill, avoiding the debilitating side effects associated with infusion centers.
Mechanism of Action and Targeted Therapy
Vorasidenib works by blocking the mutant IDH protein within cancer cells, leading to tumor shrinkage and preventing the need for more aggressive interventions. Dr. Bigner explained that this targeted approach allows for treating only patients likely to respond, improving outcomes and reducing unnecessary treatments.
Impact and Future Research
The FDA approval of vorasidenib offers new hope for patients with Grade 2 IDH-mutant gliomas. Servier Pharmaceuticals CEO David Lee emphasized the drug's potential to improve patients' quality of life, allowing them to pursue their careers and make memories with their families. Ongoing research at Duke University is exploring the use of vorasidenib in patients with more aggressive tumors or those who have undergone other therapies. Four additional clinical trials are currently underway at Duke.
According to the FDA, this approval is the first of its kind for a systemic therapy targeting Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. Vorasidenib is administered orally once daily, providing a convenient and well-tolerated treatment option for patients.
