Vorasidenib
- Generic Name
- Vorasidenib
- Drug Type
- Small Molecule
- Chemical Formula
- C14H13ClF6N6
- CAS Number
- 1644545-52-7
- Unique Ingredient Identifier
- 789Q85GA8P
Genomic Testing Emerges as Critical Tool in Brain Cancer Treatment, Experts Emphasize
• Comprehensive genomic profiling is crucial for brain cancer patients, enabling personalized treatment approaches and identification of actionable mutations like NTRK fusions, according to Dr. Manmeet Ahluwalia.
• The recent FDA approval of vorasidenib (Voranigo) for IDH-mutant gliomas represents a breakthrough in targeted therapy for brain tumors and provides hope for developing additional brain-penetrating drugs.
• Clinical trials remain the optimal treatment option for aggressive brain cancers like glioblastoma, with increasing availability in both academic centers and larger community practices.
Brain Hemorrhage Treatment Market Expected to Grow Significantly by 2032 as AI Solutions Gain FDA Approval
• The brain hemorrhage treatment market is projected to experience substantial growth through 2032, driven by an aging population and increasing prevalence of risk factors such as hypertension and traumatic brain injuries.
• Recent FDA approvals for AI-based diagnostic tools, including JLK-LVO and Heuron ICH, mark significant advancements in early detection and treatment of brain hemorrhages.
• Several pharmaceutical companies including Baxter, Pfizer, and Novartis are developing promising pipeline therapies such as tissue plasminogen activator, alteplase, and BAF312 to address the significant unmet needs in brain hemorrhage treatment.
Novel Treatment Approaches Show Promise in Brain Cancer: From Aerosolized Drugs to Personalized Vaccines
• Recent developments in brain metastases treatment showcase promising results with targeted therapies like osimertinib, offering improved blood-brain barrier penetration and more focused radiation approaches.
• Innovative treatment methods under investigation include NEO100, an aerosolized perillyl alcohol delivered nasally, being tested in meningioma and IDH-mutant glioma patients.
• A personalized vaccine approach using patients' tumor tissue is being evaluated in a phase 2b trial for glioblastoma, representing a new frontier in immunotherapy for brain cancer.
FDA Approves Voranigo: First Targeted Therapy for IDH-Mutant Glioma Brain Cancer
• The FDA has approved Voranigo, marking a historic breakthrough as the first targeted therapy for grade 2 IDH-mutant glioma, offering new hope for brain cancer patients.
• Clinical trials demonstrated that Voranigo, an oral medication, significantly delays the need for aggressive interventions like radiation, surgery, and chemotherapy in glioma patients.
• This breakthrough treatment particularly benefits younger patients in their 30s and 40s who are diagnosed with this incurable brain tumor while in the prime of their lives.
Servier to Present Case for Novel IDH-Mutant Glioma Drug Vorasidenib at EMA Review
Servier is preparing for a critical oral explanation meeting with the European Medicines Agency (EMA) regarding its innovative brain tumor therapy, vorasidenib. The drug targets Grade 2 IDH-mutant glioma, representing a potential new treatment option for this challenging form of brain cancer.
Ivy Brain Tumor Center Advances Novel Therapies for Brain Cancer
• The Ivy Brain Tumor Center is focusing on early-phase trials using pharmacokinetic and pharmacodynamic strategies to identify effective brain cancer drugs.
• The center is expanding its focus to Phase 3 studies and exploring new drug classes like PROTACs and antibody-drug conjugates for neuro-oncology.
• A Phase 0/1 trial (NCT06072586) is underway, evaluating BDTX-1535, a fourth-generation EGFR inhibitor, for recurrent high-grade glioma with EGFR alterations.
• Biomarker testing, including gene sequencing and immunohistochemistry, aids in identifying targetable alterations, exemplified by vorasidenib for IDH mutations.
Brain-Penetrant EGFR Inhibitor BDTX-1535 Shows Promise in Recurrent High-Grade Glioma Trial
• The Ivy Brain Tumor Center is conducting a phase 0/1 trial of BDTX-1535, a fourth-generation EGFR inhibitor, in patients with recurrent high-grade glioma.
• BDTX-1535 is designed to penetrate the blood-brain barrier, addressing a limitation of previous EGFR inhibitors in neuro-oncology.
• The trial assesses drug accumulation in tumor tissue and explores resistance mechanisms through cerebrospinal fluid biopsies.
• Early data focuses on identifying patient populations that benefit most and adapting treatment strategies based on real-time tumor evolution.
Agios' Pyrukynd Faces FDA Decision for Thalassemia Treatment by September 2025
• The FDA has accepted Agios' sNDA for Pyrukynd (mitapivat) to treat adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, setting a PDUFA goal date of September 7, 2025.
• Pyrukynd, a pyruvate kinase activator, could become the first oral therapy approved for all thalassemia subtypes, addressing a significant unmet need for patients with limited treatment options.
• Agios completed enrollment for the Phase 3 RISE UP study of mitapivat in sickle cell disease, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026.
Top Neurology Podcasts of 2024 Highlight Breakthroughs in MS, Alzheimer's, and ALS Research
NeurologyLive's Mind Moments podcast featured groundbreaking discussions on updated MS diagnostic criteria, advances in Alzheimer's treatment, and developments in ALS therapeutics throughout 2024. The most popular episodes covered critical topics including the new McDonald criteria for MS diagnosis, emerging RNA therapeutics for Alzheimer's disease, and insights from the PHOENIX trial in ALS.
FDA Approvals in 2024: Advancing Treatment Paradigms in Solid Tumors and Hematologic Malignancies
• The FDA granted over 65 approvals in 2024, significantly impacting treatment paradigms across various cancers, including breast, gynecologic, skin, and genitourinary malignancies.
• Several tumor-agnostic approvals, such as fam-trastuzumab deruxtecan-nxki (Enhertu) for HER2-positive solid tumors and repotrectinib (Augtyro) for NTRK fusion-positive tumors, marked advancements in precision medicine.
• Immunotherapies like nivolumab (Opdivo) and pembrolizumab (Keytruda) received multiple approvals, including combinations with chemotherapy for urothelial and endometrial carcinomas, improving patient outcomes.
• Targeted therapies like vorasidenib (Voranigo) for low-grade glioma and selpercatinib (Retevmo) for RET-mutated thyroid cancers addressed unmet needs and demonstrated high efficacy and tolerability.
MSK Researchers Spearhead Advances in Cancer Treatment, Leading to 11 FDA Approvals in 2024
• Memorial Sloan Kettering Cancer Center (MSK) made significant strides in cancer treatment in 2024, including novel vaccines, drugs enhancing radiation, and advanced diagnostics.
• A vaccine targeting KRAS mutations in pancreatic and colorectal cancers showed promising early results in stimulating the immune system.
• Imlunestrant, combined with abemaciclib, demonstrated a significant reduction in cancer progression or death in ER+, HER2- advanced breast cancer patients resistant to standard therapy.
• MSK played a pivotal role in clinical trials leading to 11 FDA approvals in 2024, spanning new uses for existing drugs, effective drug combinations, and novel targeted therapies.
Vorasidenib Shows Sustained Benefit in IDH-Mutant Low-Grade Glioma
• Vorasidenib demonstrates a significant and sustained improvement in progression-free survival (PFS) compared to placebo in adults with IDH-mutant low-grade glioma.
• The median time to next intervention (TTNI) was significantly longer with vorasidenib, indicating a durable treatment effect and delaying the need for subsequent therapies.
• Updated results from a phase I study suggest vorasidenib and ivosidenib show promising activity in diffuse IDH1-mutant glioma, with vorasidenib demonstrating a higher response rate.
• Vorasidenib's manageable safety profile and ability to reduce tumor volume, even after crossover from placebo, further support its role in treating IDH-mutant low-grade glioma.
Vorasidenib Approved in China's Boao Lecheng for IDH-Mutant Glioma Treatment
• Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
• This approval allows Chinese patients with IDH-mutant diffuse glioma access to the first-in-class targeted therapy before its official registration in China.
• A real-world study is underway in Boao Lecheng to assess the safety and efficacy of Vorasidenib in Chinese patients, potentially accelerating research by 1-2 years.
• Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, marking its global recognition as an important treatment option.
Vorasidenib Approved in China's Boao Lecheng Pilot Zone for IDH-Mutant Glioma
• Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
• This approval allows Chinese patients with IDH-mutant diffuse glioma access to the drug before its official registration in China, aligning with global availability.
• A real-world study is underway in Boao Lecheng to evaluate vorasidenib's safety and efficacy in Chinese patients, potentially accelerating research by 1-2 years.
• Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, marking a significant advancement in glioma treatment.
Vorasidenib Approved for Clinical Use in China's Boao Lecheng Pilot Zone
• Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
• This approval marks the first use of Vorasidenib in Asia, preceding its availability in Europe, for treating Chinese patients with IDH-mutant diffuse glioma.
• A real-world study is underway in Boao Lecheng to evaluate Vorasidenib's safety and efficacy in Chinese patients, potentially accelerating research by 1-2 years.
• Vorasidenib has already been approved in the US, Canada, Australia, Israel, Switzerland and UAE, and is in preparation for official registration in China.
Vorasidenib Approved for Clinical Use in China's Boao Lecheng Pilot Zone
• Vorasidenib, a dual inhibitor of IDH1 and IDH2, has been approved for clinical use in the Boao Lecheng International Medical Tourism Pilot Zone in China.
• This approval allows Chinese patients with IDH-mutant diffuse glioma access to the targeted therapy ahead of national New Drug Application approval.
• A real-world study is underway to assess Vorasidenib's safety and efficacy in Chinese patients, following approvals in the US, Canada, and other countries.
• Vorasidenib represents a significant advancement in glioma treatment, addressing a critical need for therapies targeting IDH mutations.
Vorasidenib Confirms Efficacy and Safety in IDH1/2-Mutant Diffuse Glioma
• Updated data from the phase 3 INDIGO study demonstrates vorasidenib's continued efficacy in patients with IDH1/2-mutated diffuse glioma.
• Vorasidenib significantly improved progression-free survival compared to placebo, with a 65% reduction in disease progression or death.
• The study also revealed that vorasidenib is associated with better seizure control and a reduction in tumor volume compared to placebo.
• Vorasidenib's safety profile remains consistent, supporting its use in treating grade 2 astrocytoma or oligodendroglioma with IDH1/2 mutations.
FDA Weighs Removal of Oral Phenylephrine; Approves Drugs for CML, Gastric Cancer, and UTIs
• The FDA is considering removing oral phenylephrine from its OTC Monograph due to a lack of efficacy as a nasal decongestant, potentially impacting numerous cold and allergy products.
• Novartis' Scemblix expands its FDA-approved uses to include the treatment of newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ PML).
• Astellas Pharma's Vyloy (zolbetuximab) receives FDA approval as the first treatment targeting the CLDN18.2 protein for gastric or gastroesophageal junction adenocarcinoma.
• Iterum Therapeutics' Orlynvah (sulopenem) gains FDA approval for treating uncomplicated urinary tract infections in women with limited oral antibacterial treatment options.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Recent FDA Approvals and Clinical Trial Updates in Oncology Highlighted on OncLive On Air
• Durvalumab gains FDA approval for perioperative treatment of resectable NSCLC, showing improved event-free survival in the AEGEAN trial.
• Vorasidenib receives FDA approval for IDH1/2-mutated grade 2 astrocytoma and oligodendroglioma, demonstrating significant progression-free survival benefits.
• Denileukin diftitox-cxdl is approved by the FDA for relapsed/refractory CTCL, offering a new treatment option for patients after prior systemic therapies.
Drug Development Updates
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