Vorasidenib

The authors declare no competing interests.

Agios Pharmaceuticals received orphan drug designation (ODD) from the FDA for tebapivat to treat myelodysplastic syndromes (MDS). The designation offers benefits like tax credits, fee exemptions, and market exclusivity. Agios aims to deliver the first oral therapy addressing anaemia in lower-risk MDS, affecting around 75,000-80,000 patients in key markets. The company focuses on developing therapies for rare diseases, with lead asset mitapivat, a PK activator, previously receiving ODD for PK deficiency, thalassaemia, and sickle cell disease.

OncLive’s weekly roundup highlights FDA approvals, including afamitresgene autoleucel for synovial sarcoma, vorasidenib for astrocytoma/oligodendroglioma, CB-012 for AML, and companion diagnostics for olaparib/abiraterone in BRCA+ mCRPC. Interviews discuss targeted therapies in mCRC and ASCO’s health equity recommendations.

Vorasidenib (Voranigo) received FDA approval for IDH1/2-mutant glioma treatment, marking the first systemic medication for low-grade glioma in over 20 years. The INDIGO trial showed a median progression-free survival of 27.7 months with vorasidenib vs 11.1 months with placebo. Vorasidenib is well-tolerated, with common side effects being fatigue, headache, and nausea, mostly grade 1. The approval allows for broader use, but careful patient-provider discussions are essential due to potential overtreatment risks and patient-specific considerations.

Vorasidenib, a dual inhibitor of IDH1 and IDH2 mutations, offers a breakthrough in treating IDH-mutated, grade two glioma, significantly improving progression-free survival and crossing the blood-brain barrier effectively.

Dr. Darrell Bigner's team discovered a point mutation in the isocitrate dehydrogenase gene in brain tumors, leading to the development of vorasidenib (Voranigo) by Servier Pharmaceuticals. The drug, approved by the FDA, treats one-third of primary brain tumor types and has shown promising results in clinical trials at Duke, with patients experiencing stable MRIs and no tumor progression.

Vorasidenib, a new targeted treatment for low-grade glioma, has shown promising results in a phase 3 trial, doubling progression-free survival compared to placebo. Approved by the FDA in the USA, its approval in the UK and EU is pending. The drug targets IDH1 and IDH2 mutations common in gliomas, offering a less toxic treatment option.

FDA approved vorasidenib, a targeted drug for low-grade gliomas with IDH1/2 mutations, doubling progression-free survival to 27.7 months vs. 11.1 months for placebo. Developed through Duke and Johns Hopkins collaboration, it inhibits mutant IDH enzymes, reducing tumorigenesis. Future research explores its combination with other therapies and broader cancer applications.

FDA approves Voranigo (vorasidenib), an IDH1 and IDH2 inhibitor, for treating Grade 2 IDH-mutant glioma in patients 12+ years old post-surgery. Voranigo offers a once-daily pill for disease management, with Phase 3 INDIGO trial results showing significant progression-free survival extension and safety.