MEI Pharma Inc. (Nasdaq: MEIP), an oncology company based in San Diego, has been granted an orphan drug designation for the company's investigational drug Pracinostat by the U.S. Food and Drug Administration.
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 country. Pracinostat is being investigated for use in the treatment of acute myeloid leukemia.
The drug is an orally available enzyme inhibitor, targeting histone deacetylase enzymes. It's been tested in a number of Phase 1 and Phase 2 clinical trials, MEI said, in advanced blood disorders and solid tumor indications in both adult and pediatric patients. The company said it has been generally well tolerated in more than 200 patients to date.
Acute myeloid leukemia, also known as acute myelogenous leukemia, is reportedly the most common acute leukemia affecting adults. MEI said its incidence is expected to increase as the population ages. The American Cancer Society estimates about 14,590 new cases of AML per year in the U.S., with an average new patient age of about 66.
The FDA's orphan designation qualifies MEI for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.