The FDA Has Approved TIBSOVO for ADH1-Mutated AML

Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion of its previous approvals, as it has already been given the green light for IDH1-mutated, reduced or refractory (R/R) AML; cholangiocarcinoma that is IDH1-mutated and has already been treated; and newly diagnosed IDH1-mutated AML patients ≥75 years old. You can read all about TIBSOVO’s latest approval in a press release from Servier Pharmaceuticals.

About AML

First thing’s first, let’s talk about what this rare cancer is. AML is a blood and bone marrow cancer which sees the bone marrow produce abnormal red blood cells, myeloblasts, and/or platelets. These abnormal cells go on to overcrowd the healthy cells, making it difficult for normal bodily functions to occur. This manifests as symptoms such as:

  • Fatigue
  • Bone pain
  • Fever
  • Pale skin
  • Shortness of breath
  • Easy bruising
  • Frequent infections
  • Abnormal bleeding
    • Examples include bleeding of the gums and frequent nosebleeds

Medical professionals aren’t sure why the bone marrow produces these abnormal cells, but they have identified a few risk factors: certain chemotherapy drugs, exposure to certain chemicals, and radiation.


The latest approval for TIBSOVO is based on the AGILE trial, a phase 3, randomized, double-blind, multi-center, placebo-controlled study focused on the efficacy and safety of TIBSOVO when used in combination with azacitidine. Researchers focused on the primary endpoint of event-free survival (EFS), which is defined as the time from randomization until relapse, treatment failure, or death. Secondary endpoints were also analyzed, including complete remission (CR) rate, overall survival (OS), partial remission (PR), objective response rate (ORR), morphologic leukemia-free state (MLFS), complete remission with partial hematologic recovery (CRh) and CR rate, and CR with incomplete hematologic recovery (CRi).

Results include:

  • Statistically significant improvement in EFS
  • Statistically significant improvement in OS
  • Compared to placebo plus azacitidine, TIBSOVO plus azacitidine improved the median OS by three times
    • 7.9 months vs 24 months, respectively
  • Consistent safety profile
    • The most common adverse reactions were insomnia, vomiting, nausea, leukocytosis, differentiation syndrome, hypertension, electrocardiogram QT prolonged, headaches, arthralgia, dyspnea, and hematoma
    • Laboratory abnormalities were decreases in the following: platelets, hemoglobin, leukocytes, neutrophils, phosphate, and magnesium. There were increases in glucose, lymphocytes, alkaline phosphatase, potassium, and aspartate aminotransferase

You can read the full results in the New England Journal of Medicine.

What Does this Mean for Patients?

This approval means that AML patients who were not eligible for intensive chemotherapy – who previously had few treatment options – now have a new choice. Additionally, TIBSOVO is the first therapy to target cancer metabolism that provides such positive results in EFS and OS when combined with azacitidine.

To show how much it believed this therapy would aid AML patients, the FDA granted TIBSOVO’s supplemental New Drug Application (sNDA) priority review through its Real-Time Oncology Review (RTOR) pilot program. This program was created to get effective, safe treatments to patients as soon as possible.

If you’re interested in TIBSOVO as a patient, you should reach out to your doctor to learn more. Servier also offers a program called ServierONE Patient Support Services, which provides one-on-one support for patients using a Servier drug. This support includes emotional support, financial assistance, and further resources.

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