FDA Approves Voranigo, the First Targeted Therapy for Grade 2 IDH-Mutant Glioma

Approval Date:
The FDA approved Voranigo (vorasidenib) on August 6, 2024, for the treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation in adult and pediatric patients 12 years and older.

First Targeted Therapy:
Voranigo is the first systemic therapy approved for low-grade IDH-mutant brain cancer, marking a significant advancement in the treatment of this type of brain tumor.

Clinical Trial:
The efficacy of Voranigo was evaluated in the INDIGO trial, a randomized, multicenter, double-blind, placebo-controlled study involving 331 patients. The trial demonstrated a significant improvement in progression-free survival (PFS) and time to next intervention.

Dosage:
The recommended dose for adult patients is 40 mg orally once daily. For pediatric patients 12 years and older, the dose is 40 mg for those weighing ≥ 40 kg and 20 mg for those weighing < 40 kg.

Side Effects:
Common adverse reactions include fatigue, headache, COVID-19 infection, musculoskeletal pain, diarrhea, nausea, and seizure. Serious side effects may include liver problems and changes in liver function blood tests.

Regulatory Collaboration:
The FDA collaborated with international regulatory agencies, including the Australian Therapeutic Goods Administration, Brazilian Health Regulatory Agency, Health Canada, Swissmedic, and Israel’s Ministry of Health, under Project Orbis.

Significance:
This approval represents the first major treatment advance in low-grade brain cancer in over two decades, offering patients a new option to manage their disease.