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Agios Pharmaceuticals (AGIO) has already announced plans to seek U.S. approval for its second leukemia drug by the end of the year. On Wednesday, the biotech firm provided a peek at some of the clinical trial data that will support the new filing.

The Agios drug is called ivosidenib, and it works by blocking a mutated enzyme, IDH1, that is found in 6 to 10 percent of patients with acute myeloid leukemia (AML), a type of blood cancer that forms in the bone marrow and causes unchecked growth of abnormal white blood cells.


In a clinical trial of 125 patients with relapsed or refractory AML harboring the IDH1 mutation, treatment with ivosidenib led to complete remission (CR) rate of 21.6 percent. Another 8.8 percent of patients showed a complete response with partial hematologic recovery (CRh), for a combined CR/CRh rate of 30.4 percent. Median duration of response for these patients was 8.2 months.

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  • So, if Agios is seeking end of the year filing ; is that breakthrough status ? If so, under the new FDARA Act, Race For Children , they are supposed to post expanded access policy and contact info. To allow a chance at life for those many not in their small, slow trials. Not that they shouldn’t already be doing the right moral thing , but that is lacking in PHRMA World…

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