Aug 30, 2021

Novartis announced today that the European Commission (EC) has granted orphan drug designation to sabatolimab (MBG453) for the treatment of myelodysplastic syndromes (MDS), based on clinical data showing a high rate of responses in patients with high-risk MDS who were treated with sabatolimab in combination with hypomethylating agents (HMAs). The decision follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA)1.

  • MDS are a group of rare and often underdiagnosed blood cancers characterized by two major components, a dysfunctional immune system and leukemic stem cell proliferation, which limits the ability of current treatment options to achieve durable responses2-4.
  • Patients with MDS face the possibility of poor outcomes with a limited duration of response and a median overall survival rate of less than two years despite treatment with the current standard of care5,6.
  • Sabatolimab is an investigational, potential first-in-class immuno-myeloid therapy that binds to TIM-3, a novel target expressed on immune and leukemic cells, but not on normal hematopoietic stem cells. It is in development for the treatment of higher-risk MDS and acute myeloid leukemia (AML). Targeting TIM-3 reawakens the immune system to selectively attack leukemic stem cells, the source of MDS/AML, and has the potential to safely deliver a durable response7,8.

Orphan drug designation is reserved for medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease with a prevalence in the EU of below 5 in 10,000 and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease9. At the time of the EMA’s evaluation of the marketing authorization application for sabatolimab, the COMP will determine whether the orphan designation can be maintained based on an analysis of available data1.

The EC decision follows the US Food and Drug Administration’s fast track designation for sabatolimab in May 2021, for the treatment of adult patients with MDS defined with an IPSS-R risk category of high- or very high-risk in combination with HMAs1.

  1. Novartis data on file
  2. Brunner AM, et al. Recent advances in the cellular and molecular understanding of myelodysplastic syndromes: implications for new therapeutic approaches. Clin Adv Hematol Oncol. 2018 Jan;16(1):56-66.
  3. Steensma, DP, et al. Myelodysplastic Syndromes: Diagnosis and Treatment. Mayo Clin Proc. July 2015;90(7):969-983.
  4. Glenthøj A, et al. Immune Mechanisms in Myelodysplastic Syndrome. Int J Mol Sci. 2016 Jun; 17(6): 944.
  5. Stein EM, et al. Treatment patterns and outcomes in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis. Leuk Lymphoma. 2021 Jan 11:1-16.
  6. Faber MG, et al. Current state of myelodysplastic syndromes: standard treatment practices and therapeutic advances. J Clin Pathways. 2019;5(6):43-47.
  7. Borate, U., et al. Phase Ib Study of the Anti-TIM-3 Antibody MBG453 in Combination with Decitabine in Patients with High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML). Blood (2019) 134(1): 570.
  8. Wolf Y., et al. TIM3 comes of age as an inhibitory receptor. Nat Rev Immunol. 2020 Mar 20(3):173-185.
  9. European Medicines Agency. Orphan Designation Overview. Accessed August 2021. Available at:…


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