FDA Approves Novel Therapy for Rare Form of Blood Cancers

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The treatment has received Priority Review designation, as well as FDA Breakthrough Therapy designation and Orphan Drug designation.

On Oct 24, 2023 FDA approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) for the treatment of adults with “relapsed or refractory (R/R) myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.” Ivosidenib is the first targeted therapy approved for this indication. Abbott Laboratories’ RealTime IDH1 was also approved as a companion diagnostic for the selection of R/R MDS patients with an IDH1 mutation.

MDS are a rare form of blood cancers that develop when mutations in bone marrow progenitor cells (cells that form blood) cause inadequate numbers of healthy blood cells. Approximately 60,000 to 170,000 people in the United States suffer from MDS, and about 87,000 new cases are reported each year worldwide. Around 3.6% of MDS patients have an IDH1 mutation.

This new indication is based on an open-label, single-arm, multicenter study of 18 adult patients with relapsed or refractory MDS with an IDH1 mutation. The mutations were found in peripheral blood or bone marrow via a local or central diagnostic test and were later confirmed with the Abbott RealTime IDH1 Assay. The complete or partial remission rate was 39% (7/18), and of the patients who achieved a complete remission, the average time to complete remission was 1.9 months. Of the nine patients who relied on blood or platelet transfusions, six no longer required transfusions after treatment with ivosidenib.


Ivosidenib was previously approved for select adults with freshly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, and locally advanced or metastatic cholangiocarcinoma. The Abbott RealTime IDH1 was also approved as a companion diagnostic to identify AML patients with an IDH1 mutation for treatment with ivosidenib or Rezlidhia (olutasidenib). Ivosidenib was granted Priority Review designation, under which FDA is prepared to act on an application within six months of the agency determining that the drug would substantially improve the safety or efficacy of treating, diagnosing, or preventing a serious condition. Ivosidenib also received FDA Breakthrough Therapy designation and Orphan Drug designation.

Source: FDA