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Cancer is the second leading cause of death in the U.S.1 To address the growing need for therapeutic solutions, Servier has made Oncology one of our priority innovation areas. Servier is the leader in IDH-mutant targeted therapies. Guided by patient needs, we are focused on developing major therapeutic innovations for rare cancers with high unmet medical needs.

Gliomas are tumors that arise from certain types of brain or spinal cord cells.2

  • Adult-Type Diffuse Gliomas

There are multiple different groups of gliomas, one of which is adult-type diffuse gliomas.3 These diffuse gliomas are the most common primary malignant brain tumors in adults.3

Genetic mutations have an impact on the glioma classification and potential prognosis.4 Mutated IDH gliomas account for about 20% of adult-type diffuse glioma.4 Each year, approximately 2,400 people are diagnosed with IDH-mutant glioma in the U.S. alone.4 Molecular testing is required for proper diagnosis.5

  • Pediatric Low-Grade Glioma

Pediatric low-grade gliomas (pLGG) are the most common brain tumors in children, accounting for approximately 30% of all childhood central nervous system tumors in the U.S.6 pLGG tumors are generally slow-growing with high survival rates, though patients can experience ongoing disease burden that may persist throughout life.6

Alterations in the BRAF gene are detected in up to 75% of pediatric low-grade astrocytomas and can drive tumor proliferation in pLGGs.7,8

Acute myeloid leukemia (AML) is a cancer of blood and bone marrow.9 AML is characterized by rapid disease progression and is one of the most common acute leukemias affecting adults,9 with approximately 22,000 new cases in the U.S. each year.10 For 6-10% of AML patients, the mutated IDH1 enzyme blocks normal blood stem cell differentiation, contributing to the genesis of acute myeloid leukemia.11

Myelodysplastic Syndromes (MDS) are disorders in which stem cells do not mature into healthy blood cells.12 In the U.S., approximately 10,000-15,000 new cases of MDS are reported each year.13 Approximately 3.6% of MDS patients have an IDH1 mutation,14 which is considered an early “driver” mutation.15 For MDS patients with an IDH1 mutation, prognosis is often associated with worse overall outcomes and in an increased risk of transformation to AML.14

Acute Lymphoblastic Leukemia (ALL) is a rare, rapidly progressing blood cancer that starts in the bone marrow and spreads into the blood.16,17 Most cases of ALL occur in children, but can occur in adults as well.17 ALL represents about 75-80% of childhood leukemia cases.18

Cholangiocarcinoma (CCA), also known as bile duct cancer, is a rare and aggressive type of cancer that affects the bile ducts, which carry digestive fluid (bile) and connect the liver, gallbladder and small intestine.19

Approximately, 8,000 people in the U.S. are diagnosed with CCA each year, but the actual number of cases may be higher.20 The disease can be hard to diagnose, leading CCA to be misclassified as other cancers.20

IDH1 mutations occur in up to 25% of patients with cholangiocarcinoma.21


References

  1. U.S. Centers for Disease Control. Chronic Disease Indicators. Cancer. Available at: link. Accessed April 2026.  
  2. Cleveland Clinic. Glioma. Available at: link. Accessed April 2026.
  3. Louis DN, et al. Neuro Oncol. 2021. https://doi.org/10.1093/neuonc/noab106
  4. End Brain Cancer Initiative. Glioma Study.  Available at: link. Accessed April 2026.
  5. Alnahhas, I. Curr Oncol Rep. 2024. https://doi.org/10.1007/s11912-024-01602-w.
  6. Zelt, S, et al. Neuro-Oncology Practice. 2024. https://doi.org/10.1093/nop/npae037.
  7. Sun Y, et al. Neuro Oncol. 2017.  https://doi.org/10.1093/neuonc/now261.
  8. Schaff LR, et al. Am Soc Clin Oncol Educ Book. 2024. https://doi.org/10.1200/EDBK_431450.
  9. American Cancer Society. What is Acute Myeloid Leukemia? Available at: link. Accessed April 2026.
  10. National Cancer Institute Surveillance, Epidemiology, and End Results Program. Cancer Stat Facts: Acute Myeloid Leukemia (AML). Available at: link. Accessed April 2026.
  11. DiNardo C. New England Journal of Medicine. 2018. https://doi.org/10.1056/NEJMoa1716984.
  12. Pagliuca, S., et al. Cancers. 2021. https://doi.org/10.3390/cancers13040784.
  13. MDS Foundation. MDS By the Numbers. Available at: link. Accessed April 2026.
  14. Thol, F., et al. Haematologica. 2010. https://doi.org/10.3324/haematol.2010.025494.
  15. DiNardo CD, et al. Leukemia. 2016. https://doi.org/10.1038/leu.2015.211
  16. American Cancer Society. About Acute Lymphocytic Leukemia (ALL). Available at: link. Accessed April 2026.
  17. American Cancer Society. Key Statistics for Acute Lymphocytic Leukemia (ALL). Available at: link. Accessed April 2026.
  18. Dana-Farber Cancer Institute. Childhood Acute Lymphoblastic Leukemia. Available at: link. Accessed April 2026.
  19. Cleveland Clinic. Cholangiocarcinoma. Available at: link. Accessed April 2026.
  20. American Cancer Society. Key Statistics for Bile Duct Cancer. Available at: link. Accessed April 2026.
  21. Su, L., et al. Front Endocrinol. 2020. https://doi.org/10.3389/fendo.2020.00189