Regulatory Affairs

Agios Pharmaceuticals secures FDA nod for leukemia treatment Tibsovo

PBR Staff Writer Published 23 July 2018

Agios Pharmaceuticals has secured approval from the US Food and Drug Administration (FDA) for its Tibsovo (ivosidenib) to treat adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation.

Tibsovo, which is an oral and targeted inhibitor of the IDH1 enzyme, is said to be the first and only FDA-approved therapy for patients with R/R AML and an IDH1 mutation.

AML is a cancer of the blood and bone marrow, and most common acute leukemia affecting adults with around 20,000 new cases estimated in the US every year.

Agios secured FDA approval for Tibsovo based on the clinical data from an open-label, single-arm and multicenter dose-escalation and expansion trial of adult patients with R/R AML and an IDH1 mutation.

Tibsovo has been approved concurrently with the Abbott RealTime IDH1 companion diagnostic test for selection of patients with R/R AML for treatment with Agios’ oral therapy.

Agios assessed the efficacy of Tibsovo in 174 adult patients with R/R AML with an IDH1 mutation identified or confirmed by the Abbott RealTime IDH1 assay.

Tibsovo was administered orally at a starting dose of 500mg daily until disease progression, development of unacceptable toxicity, or undergoing hematopoietic stem cell transplantation.

According to the company, the primary endpoint is the combined complete remission (CR) and complete remission with partial hematologic improvement (CRh) rate.

Agios Pharmaceuticals CEO Dr David Schenkein said: “I want to thank the patients and their caregivers, nurses and physicians who participated in our clinical trials.

“With their support and the dedication of Agios’ employees, we are well on our way to becoming a sustainable multi-product biopharmaceutical company delivering medicines that have the potential to change how serious diseases are treated.”

Agios is engaged in the discovery and development of novel investigational medicines to treat cancer and rare genetic diseases.

The firm has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development.

Image: Tibsovo 250mg bottle. Photo: courtesy of Agios, Inc.