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FDA Approves Drug Designed to Target Specific Mutation in Rare Blood Cancer Type

The Leukemia & Lymphoma Society has provided funding to research that identified novel ways to attack the IDH mutation in patients with blood cancers

Rye Brook, N.Y., November 6, 2023 – The U.S. Food and Drug Administration (FDA) recently approved ivosidenib (Tibsovo®) for use in certain adults with myelodysplastic syndromes (MDS), which are a rare form of blood cancers. 

This approval is for patients whose MDS has an IDH1 mutation and has returned after prior successful treatment or has stopped responding to active treatment. Ivosidenib is a form of precision medicine designed to inhibit a specific mutated protein called IDH1. 

“LLS has a long-standing history of supporting the development of precision medicines, as well as research that defined this pathway and novel ways to target the IDH mutation,” said Lee Greenberger, Ph.D., Chief Scientific Officer at The Leukemia & Lymphoma Society.

By attacking cancer cells while leaving healthy cells alone, precision medicine helps to avoid short- and long-term side effects of conventional “one-size-fits-all” chemotherapies.

Approximately 40% of patients enrolled on to the trial that led to this approval achieved some form of treatment response. Moreover, six of the nine patients who needed blood or platelet transfusions because of their cancer at the beginning of the study no longer needed transfusions after receiving treatment. 

Ivosidenib is also approved for use in certain adults with newly diagnosed acute myeloid leukemia (AML), as well as previously treated AML. 

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