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Disease

Astrocytoma

Late-stage therapeutic developmentEmerging researchRising momentum
5
Publications
19
Clinical trials
3
Related conditions
1
Related proteins
2025
Latest publication
Current focus
Isocitrate dehydrogenase biologyTherapeutic developmentGenetics & risk factors
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

FDA Approval Summary: Vorasidenib for IDH-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery.

Research2025-11-01Clinical cancer research : an official journal of the American Association for Cancer Research

Approval: Voranigo (EMA)

Regulatory2025-09-17EMA

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Major developments
Important regulatory approvalImportant
Voranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy (see section 5.1).2025-09-17
Research Highlights1View
Clinical Milestones8View
Regulatory Updates1View
  • 2025-09-17Approval — VorasidenibVoranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy (see section 5.1).
Activity timeline10

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Vorasidenibapproved

Approval — Voranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Gra… (2025)

Carmustineapproved

Approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)

Everolimusapproved

Approval — Renal angiomyolipoma associated with tuberous sclerosis complex (TSC) Votubia is indicate… (2011)

Temozolomideapproved

Approval — Temozolomide Sun is indicated for the treatment of: adult patients with newly diagnosed… (2011)

Clinical trials

15 sponsors · 2 new · 1 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
9
Late-stage
6
Completed
Late-stage studies
Recruiting
Recently completed

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2025emaApprovalVorasidenib· Voranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy (see section 5.1). source ↗
2018emaApprovalCarmustine· Carmustine is indicated n adults in the following malignant neoplasms as a single agent or in combination with other antineoplastic agents and/or other therapeutic measures (radiotherapy, surgery): Brain tumours (glioblastoma, brain-stem gliomas, medulloblastoma, astrocytoma and ependymoma), brain metastases Secondary therapy in non-Hodgkin’s lymphoma and Hodgkin’s disease as conditioning treatment prior to autologous haematopoietic progenitor cell transplantation (HPCT) in malignant haematological diseases (Hodgkin’s disease / Non-hodgkin’s lymphoma). source ↗
2011emaApprovalEverolimus· Renal angiomyolipoma associated with tuberous sclerosis complex (TSC) Votubia is indicated for the treatment of adult patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC) who are at risk of complications (based on factors such as tumour size or presence of aneurysm, or presence of multiple or bilateral tumours) but who do not require immediate surgery. The evidence is based on analysis of change in sum of angiomyolipoma volume. Subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) Votubia is indicated for the treatment of patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) who require therapeutic intervention but are not amenable to surgery. The evidence is based on analysis of change in SEGA volume. Further clinical benefit, such as improvement in disease?related symptoms, has not been demonstrated. source ↗
2011emaApprovalTemozolomide· Temozolomide Sun is indicated for the treatment of: adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy (RT) and subsequently as monotherapy treatment; children from the age of three years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy. source ↗
2010emaApprovalTemozolomide· For the treatment of adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy (RT) and subsequently as monotherapy treatment. For the treatment of children from the age of three years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy. source ↗
2010emaApprovalTemozolomide· For the treatment of adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy (RT) and subsequently as monotherapy treatment. For the treatment of children from the age of three years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy. source ↗
1999emaApprovalTemozolomide· Temodal hard capsules is indicated for the treatment of: adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy and subsequently as monotherapy treatment; children from the age of three years, adolescents and adult patients with malignant glioma, such as glioblastoma multiforme or anaplastic astrocytoma, showing recurrence or progression after standard therapy. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

5 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20072025
Most influential

IDH1 and IDH2 mutations in gliomas.

The New England journal of medicine · 2009 · 4,526 cites

Central Nervous System Cancers, Version 3.2020, NCCN Clinical Practice Guidelines in Oncology.

Journal of the National Comprehensive Cancer Network : JNCCN · 2020 · 379 cites

Pediatric low-grade glioma in the era of molecular diagnostics.

Acta neuropathologica communications · 2020 · 258 cites

FDA Approval Summary: Vorasidenib for IDH-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery.

Clinical cancer research : an official journal of the American Association for Cancer Research · 2025 · 2 cites
Recent publications

FDA Approval Summary: Vorasidenib for IDH-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery.

Clinical cancer research : an official journal of the American Association for Cancer Research · 2025 · 2 cites

Central Nervous System Cancers, Version 3.2020, NCCN Clinical Practice Guidelines in Oncology.

Journal of the National Comprehensive Cancer Network : JNCCN · 2020 · 379 cites

Pediatric low-grade glioma in the era of molecular diagnostics.

Acta neuropathologica communications · 2020 · 258 cites

IDH1 and IDH2 mutations in gliomas.

The New England journal of medicine · 2009 · 4,526 cites
Major themes8
  • Astrocytoma2
  • Brain Neoplasms2
  • Mutation2
  • Central Nervous System Neoplasms1
  • Glioma1
  • Isocitrate Dehydrogenase1
  • Oligodendroglioma1
  • Triazines1
Leading journals5
  • Acta neuropathologica communications1
  • Clinical cancer research : an official journal of the American Association for Cancer Research1
  • Genes & development1
  • Journal of the National Comprehensive Cancer Network : JNCCN1
  • The New England journal of medicine1
Leading researchers8
  • Ahluwalia M1
  • Ananthula S1
  • Aungst SL1
  • Bachoo RM1
  • Baehring J1
  • Barbato MI1
  • Barone AK1
  • Batinic-Haberle I1
Affiliations (unnormalised)6
  • 10Roswell Park Comprehensive Cancer Center.1
  • 11Moffitt Cancer Center.1
  • 12UC San Diego Moores Cancer Center.1
  • 13Robert H. Lurie Comprehensive Cancer Center of Northwestern University.1
  • 14University of Michigan Rogel Cancer Center.1
  • 15Memorial Sloan Kettering Cancer Center.1

Disease biology

1 match

Key proteins & gene products studied in this disease. Number shows shared papers.

Related conditions

3 matches

Diseases frequently studied alongside this one. Number shows shared papers.

Disease profile

A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.

Overview

Astrocytoma is a neoplasm of the brain and spinal cord derived from glial cells. It ranges from histologically benign forms to highly anaplastic and malignant tumors, with fibrillary astrocytomas being the most common type. These tumors are classified in increasing order of malignancy from grades I through IV, and their typical location varies by age.

Causes

The supplied grounding does not support a specific external cause or aetiology for astrocytoma. It does indicate that the disease is associated with genetic alterations, including changes involving isocitrate dehydrogenase in the literature. For malignant astrocytic gliomas, the literature emphasizes underlying genetics and biology rather than a single known cause.

Pathophysiology

Astrocytomas arise from glial cells and can progress from lower-grade to highly malignant disease. Malignant astrocytic gliomas are characterized by widespread invasion throughout the brain, destruction of normal brain tissue, and resistance to traditional and newer targeted therapies. The literature also highlights genetic and signaling abnormalities, including isocitrate dehydrogenase-related changes, as part of the disease biology.

Risk factors

The grounding does not provide established risk factors for astrocytoma. It does note age-related patterns of origin, with tumors in the first two decades of life tending to arise in the cerebellar hemispheres and adult tumors more often arising in the cerebrum. In pediatric low-grade glioma, incomplete surgical resection is associated with repeat progression and morbidity, but this is a factor in clinical course rather than a proven risk factor for developing the disease.

Current standard of care

Management is described at the level of surgery, therapy, and drug therapy. The literature supports surgical treatment and, for some tumors, targeted therapeutics aimed at molecular pathways such as RAS/MAPK, particularly in pediatric low-grade glioma. For malignant astrocytic gliomas, treatment remains challenging because of therapeutic resistance, and the literature points to ongoing development of novel therapies rather than a single definitive standard drug class.

AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Neoplasms of the brain and spinal cord derived from glial cells which vary from histologically benign forms to highly anaplastic and malignant tumors. Fibrillary astrocytomas are the most common type and may be classified in order of increasing malignancy (grades I through IV). In the first two decades of life, astrocytomas tend to originate in the cerebellar hemispheres; in adults, they most frequently arise in the cerebrum and frequently undergo malignant transformation. (From Devita et al., Cancer: Principles and Practice of Oncology, 5th ed, pp2013-7; Holland et al., Cancer Medicine, 3d ed, p1082)

Identifiers
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.