This article is more than 5 years old. Information may no longer be current.
FDA grants orphan drug designation to SY-1425 for acute myeloid leukemia
Click Here to Manage Email Alerts
The FDA granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia, according to the drug’s manufacturer.
SY-1425 (Syros Pharmaceuticals) is an oral, first-in-class selective retinoic acid receptor alpha agonist.
“Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options,” David A. Roth, MD, chief medical officer of Syros, said in a company-issued press release. “We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes.”
An ongoing phase 2 trial will evaluate the safety and efficacy of SY-1425 monotherapy in genomically defined subsets of patients with AML and myelodysplastic syndrome, as well as in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.
“Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425,” Roth said. “We’re pleased with the continued progress of the ongoing phase 2 clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year.”
The FDA 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.
The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.