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news.Agios Pharmaceuticals has secured orphan drug designation from the US Food and Drug Administration (FDA) for its oral, first-in-class IDH1 mutant inhibitor, AG-120, to treat patients with acute myelogenous leukemia (AML), a cancer of blood and bone marrow.
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Currently, AG-120 is being evaluated in a Phase I clinical trial in patients with advanced hematologic malignancies that carry an IDH1 mutation.
AML is characterized by rapid disease progression, and is the most common acute leukemia in adults.
Agios chief medical officer Chris Bowden said: "Receiving orphan drug designation for AG-120 is an important milestone as we continue to move this program to late-stage development.
"We are pleased with the progress we are making in the clinic and look forward to presenting new data from our ongoing Phase I study of AG-120 at the Congress of the European Hematology Association later this week.
"We believe that AG-120, which is on track to initiate multiple expansion cohorts in the next month, has the potential to play a significant role in shifting the treatment paradigm for IDH1-mutant positive hematologic cancers from the conventional chemotherapy approach."
In the US, less than 10% of patients are eligible for bone marrow transplant, and the vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML.
The five-year survival rate for AML is about 20 to 25% and IDH1 mutations are present in about 6-10% of AML cases.