Pharma, BioPharma

FDA approves Agios Pharmaceuticals’ Tibsovo for acute myeloid leukemia

The approval is the latest of several drugs approved for the disease, a form of blood cancer, since last year.

Red seal and imprint "FDA APPROVED" on white surface. FDA - Food and Drug Administration is a federal agency of the United States Department of Health and Human Services.

The Food and Drug Administration has approved the second drug for a type of leukemia from a Cambridge, Massachusetts-based company.

Agios Pharmaceuticals said Friday that the FDA had approved Tibsovo (ivosidenib), for adults with relapsed/refractory acute myeloid leukemia that carries the mutation IDH1. The FDA had previously approved Agios’s Idhifa (enasidenib), for IDH2-mutant AML, in August 2017.

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The FDA’s Friday decision is the latest in a string of approvals by the agency since last year of drugs to treat AML, a blood cancer that mostly affects older adults and whose drug treatment, until recently, saw few new approvals and relied largely on old chemotherapy agents. Still, when patients relapse or are refractory to treatment, they have few, if any, options, Agios’s press release quoted Dr. Hagop Kantarjian, chairman of the leukemia department at University of Texas MD Anderson Cancer Center in Houston, as saying.

In April 2017, the FDA approved Novartis’ Rydapt, used to treat AML with FLT3 mutations. The agency is likewise expected to rule by Nov. 29 on whether to approve another FLT3 inhibitor, Astellas Pharma’s gilteritinib, which is designed to be significantly more potent than Rydapt. In August 2017, the FDA approved Jazz Pharmaceuticals’ Vyxeos, which combines the standard chemotherapy drugs daunorubicin and cytarabine into a single infusion. And a month later, it revived Pfizer’s Mylotarg, which combines a drug molecule with a monoclonal antibody targeting CD33, an antigen protein expressed on the surface of AML cells. Mylotarg had originally received approval in 2000, but was voluntarily withdrawn by the company in 2010 amid safety and efficacy concerns. However, subsequent studies have reaffirmed its therapeutic profile.

Tibsovo’s approval was based on results of a single-arm Phase I study, in which 174 patients were evaluable for efficacy. Among those patients, the rate of complete remissions (CR) and CR with partial hematological recovery (CRh) was 32.8 percent, while the rate of CR alone was 24.7 percent. The median CR and CRh duration was 8.2 months. The drug’s label includes a boxed warning for differentiation syndrome, a potentially fatal side effect. Among 179 patients evaluated for safety, 19 percent experienced the side effect

The drug is being marketed with a companion diagnostic to detect IDH1 mutations made by AbbVie, which also markets the IDH2-detecting companion diagnostic for Idhifa. Idhifa, whose development Agios partnered with Celgene, was approved on a Phase I/II study. Reuters reported that the monthly wholesale acquisition cost for Idhifa would be $24,872.

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