Agios Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development for its AG-221, which is used for treating patients suffering with acute myelogenous leukemia (AML) disease.

The AML is a cancer of blood and bone marrow characterised by rapid disease progression, which leads to IDH2 mutations.

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AG-221 is an IDH2 mutant inhibitor that is currently being tested in Phase I clinical trial in patients with advanced hematologic malignancies that carry an IDH2 mutation.

"AG-221 has the potential to play a significant role in shifting the treatment paradigm for IDH2 mutant positive hematologic cancers from the conventional chemotherapy approach."

According to the American Cancer Society, the median age for getting AML is 66. In addition, fewer than 10% of patients are eligible for bone marrow transplant and majority of patients do not respond to chemotherapy, leading to relapsed or refractory AML.

AML prevalence is estimated to be around 35,000 to 40,000 patients in the US, with around 15% patients estimated to carry an IDH2 mutation.

The orphan drug designation will allow Agios and its collaborator Celgene to market the product, which is subject to receipt of regulatory approval, exemption of FDA application fees and tax credits for qualified clinical trials.

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Agios Pharmaceuticals MD chief medical officer Chris Bowden said: "We are pleased with the progress we are making in the clinic and believe that AG-221, which is expected to enter multiple expansion cohorts in the second half of this year, has the potential to play a significant role in shifting the treatment paradigm for IDH2 mutant positive hematologic cancers from the conventional chemotherapy approach."