Vorasidenib

Gliomas, common brain tumors in adults, often have IDH gene mutations. Molecular testing identifies these mutations, enabling targeted treatments like IDH inhibitors, improving patient outcomes. Testing is crucial for personalized treatment and is covered by insurance.

Gliomas, common malignant brain tumors, originate from glial cells. IDH mutations fuel tumor growth; drugs targeting IDH mutations like Vorasidenib offer new treatment options, especially for low-grade gliomas. Continued research aims to uncover more gene mutations driving tumor growth for improved treatments.

FDA approved vorasidenib (Voranigo) for IDH-positive grade 2 astrocytoma or oligodendroglioma patients, based on INDIGO trial results. Dr. Jennie W. Taylor discussed this in an OncLive On Air podcast.

The FDA approved 16 oncology therapies in Q3 2024, including first-in-class TCR T-cell therapy afami-cel for synovial cell sarcoma, denileukin diftitox for cutaneous T-cell lymphoma, and axatilimab-csfr for chronic graft-versus-host disease. Vorasidenib was approved for grade 2 astrocytoma or oligodendroglioma, and three EGFR-targeting therapies for non-small cell lung cancer. New formulations of atezolizumab and daratumumab with hyaluronidase were approved, along with expanded indications for durvalumab, isatuximab, and ribociclib. Selpercatinib was approved for pediatric medullary thyroid cancer, and pembrolizumab and dostarlimab-gxly received biomarker-agnostic approvals for malignant pleural mesothelioma and endometrial cancer, respectively.

Biologics by McKesson selected by Servier as a limited specialty pharmacy provider for VORANIGO (vorasidenib), a targeted drug for Grade 2 IDH-mutant gliomas, approved by the FDA on August 6, 2024.

Drs. Lazarev and Sindhu critique vorasidenib, an FDA-approved brain tumor drug, questioning its approval process and benefits, highlighting ethical concerns and lack of clear survival or quality of life improvements compared to existing treatments.

Voranigo (vorasidenib), an IDH1/IDH2 inhibitor by Servier Pharmaceuticals, was approved by the FDA in August for treating grade 2 astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations. A phase 3 trial showed Voranigo doubled progression-free survival to 27.7 months compared to 11.1 months with a placebo.

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.