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FDA grants orphan drug designation to AG-120 for AML
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The FDA granted orphan drug designation to AG-120 for the treatment of patients with acute myelogenous leukemia, according to a press release from the drug’s manufacturer.
AG-120 (Agios) — an oral, first-in-class IDH1 mutant inhibitor — is currently under evaluation in a phase 1 study of patients with advanced hematologic malignancies who harbor an IDH1 mutation. Researchers presented data from this ongoing study at the Congress of the European Hematology Association in Vienna.
AG-120 demonstrated durable clinical activity and a favorable safety profile in 57 patients, according to the press release. The overall response rate was 31% and the complete remission rate was 15%. Further, responding patients have remained on treatment for up to 11 months, and 79% of responders received treatment for at least 3 months.
The most common adverse events — the majority of which were mild to moderate in nature — were fatigue, diarrhea, pyrexia and nausea. Thirty-five serious adverse events occurred, most of which were disease related. Four patients experienced leukocytosis, which researchers noted may have been related to study treatment.
Researchers reported 13 deaths, but none were considered related to AG-120.
“The durable clinical activity observed with AG-120 in such a refractory patient population is impressive,” Stéphane de Botton, MD, the study’s principal investigator at the Institut de Cancérologie Gustave Roussy in Villejuif, France, said in a press release. “These findings provide additional evidence that AG-120 can inhibit the IDH1-mutant protein allowing for cancer cells to appropriately mature. AG-120 has the potential to improve outcomes in patients with IDH1-mutant cancers.”
Agios plans to initiate three expansion cohorts to evaluate AG-120 in 175 patients with IDH1-mutated advanced hematologic malignancies, including relapsed and/or refractory AML, previously untreated AML and basket IDH1-mutant positive cancers
The FDA granted fast track designation to AG-120 in May.
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.