Ivosidenib Approved For Treating Relapse Or Refractory MDS With An IDH1 Mutation

The IDH1 mutation occurs in approximately 3% of MDS patients. This mutation also increases the risk of MDS developing into AML. Current treatment options include hypomethylating agents or immunosuppressive therapy. The IDH1 mutation is found in both low-risk and high-risk MDS.

Ivosidenib (Tibsovo) is an oral medication that is an IDH1 inhibitor. On October 24th 2023, the Food and Drug Administration (FDA) approved ivosidenib for treatment for patients with relapsed or refractory MDS with the IDH1 mutation.

Identifying mutations in MDS is “crucial given the large unmet clinical need in this patient population which may assist identifying potential targeted therapy.” Thus, oral inhibitors of common mutations are being explored. Ivosidenib has proven in a recent clinical trial that 75% of patients achieved overall response rate (ORR) for a duration of 21.4 months.

A Few Things To Know About Treatment With Ivosidenib:

• 18% of patients with severe neutropenia (SN) were found to have the IDH1 mutation.
• There was no difference in response rate when azacitidine was administered.
• Lower-risk patients treated with ivosidenib had increased hemoglobin rates and platelets improved.
• IDH1 patients with severe neutropenia showed that “treatment with ivosidenib resulted in ongoing, durable complete hematologic responses.”
• Ivosidenib monotherapy was tolerable and induced remission and transfusion independence.

Side Effects Can Occur When Treated with ivosidenib:

• Diarrhea, nausea, vomiting.
• Increased blood sugar.
• Fatigue and shortness of breath.
• Swelling in the arms or legs.
• Decreased appetite.
• Joint pain.
• Pain or sores in your mouth or throat.

“Ivosidenib was well tolerated in MDS patients with significant responses in all the cohorts. With a response rate of 91%, ivosidenib was particularly effective in treatment of naïve higher risk MDS patients with IDH1 mutations. These encouraging preliminary results have to be confirmed in more patients.”

The approval of ivosidenib is an exciting advancement for the MDS community. Every new approval brings us better options for patients and moves us closer to an MDS cure. We celebrate this approval and wait excitedly to see what is coming next in the treatment landscape for MDS.