Is Ivosidenib effective in treatment?

On December 26, 2017, Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leading company dedicated to treating cancer and rare genetic diseases by leveraging cellular metabolic mechanisms, announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA).

Is Ivosidenib effective in treatment?

Dr. Chris Bowden, Chief Medical Officer of Agios Pharmaceuticals, stated: "The currently available treatment options are extremely limited for patients with relapsed or refractory acute myeloid leukemia (AML), and we aim to change this situation. Earlier this month, at the Annual Meeting of the American Society of Hematology (ASH), we presented encouraging clinical data of ivosidenib as a monotherapy, demonstrating that the drug can induce durable therapeutic responses in high-risk patients with relapsed or refractory AML harboring IDH1 mutations. These data highlight the potential of ivosidenib to become the first-in-class therapy for the treatment of relapsed/refractory AML patients with IDH1 mutations."

In addition, Agios Pharmaceuticals announced that the FDA has completed the 30-day safety review of the Investigational New Drug (IND) application for its drug candidate AG-270, a therapy targeting MTAP-deficient tumors, and has approved the initiation of the proposed Phase I dose-escalation clinical trial. This trial will enroll patients with multiple tumor types harboring MTAP deletion. Agios expects to initiate this Phase I clinical trial in the first quarter of 2018.

What is Ivosidenib?

Ivosidenib is indicated for the treatment of cholangiocarcinoma, as well as certain blood and bone marrow cancers. It exerts its effect by blocking a protein that promotes cancer cell growth and proliferation, thereby helping to slow down or prevent the spread of cancer cells.

This medication may also be used for other purposes; if you have any questions, please consult your healthcare provider or pharmacist.

What information should I inform my healthcare team before taking this medication?

You need to inform them if you have any of the following conditions:

Whether you have a history of Guillain-Barré syndrome, heart disease, cardiac arrhythmia, kidney or liver disease, low blood levels of sodium, potassium or magnesium, or other neurological disorders; also state whether you have had abnormal reactions or a history of allergies to ivosidenib, any other medications, foods, dyes or preservatives; if you are pregnant, planning to become pregnant or currently breastfeeding, you should also inform your healthcare providers in advance.