The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational drug pracinostat for the treatment of acute myeloid leukemia (AML).
The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States.
Pracinostat is an orally available histone deacetylase (HDAC) inhibitor that has been tested in phase I and phase II clinical trials in advanced hematologic disorders and solid tumors in both adult and pediatric patients. The drug has been generally well tolerated, with manageable side effects generally associated the drugs of this class.
In a phase I dose-escalation trial, pracinostat demonstrated evidence of single-agent activity in elderly patients with AML, including 2 out of 14 (14%) who achieved a complete remission, with durable responses persisting 206+ and 362 days, respectively. A phase II clinical trial of pracinostat in combination with Vidaza in elderly patients with newly diagnosed AML is currently underway. ■