Ellipses Pharma’s Acute Myeloid Leukemia Candidate Gets Orphan Drug Nod

The FDA has granted Orphan Drug status to Ellipses Pharma’s EP0042, an investigational drug for treatment of patients with acute myeloid leukemia.

The drug candidate is being evaluated for patients with resistance to FLT-3 inhibitors. Approximately one-third of patients have FLT-3 mutations, which are associated with higher risk of relapse.

The drug is currently in a phase 1/2 study and the company also plans to study the drug as a potential monotherapy and in combination with other treatments when a phase 2 dose is confirmed.

The Orphan Drug Designation is given to drugs that show promising early clinical data in rare diseases that affect fewer than 200,000 people in the U.S., including acute myeloid leukemia.