Update Pharma's UPI-928 gets FDA orphan drug designation for AML treatment

Feb 18 2014

Update Pharma Inc. today announced that the FDA has approved Orphan Drug Status for the use of UPI-928 for the treatment of Acute Myeloid Leukemia (AML).

The FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the U.S. The first NDA applicant to receive FDA approval for a particular active ingredient to treat a particular disease with FDA orphan drug designation is entitled to a seven-year exclusive marketing period in the U.S. for the use of that product in the specified indication. The company may also be eligible to receive clinical trial tax incentives (21 CFR 316.20 and 316.21).

"The data for UPI-928 in AML was the basis for the approval of this agent in France around 25 years ago and compares quite favorably with the newer agents in development for this indication," mentioned John Rothman, Ph.D., CEO of Update Pharma Inc. "UPI-928 is an anthracene that lacks the lifetime dosage limit associated with other anthracyclines and has been useful in the treatment of refractory or recurrent patients who were heavily pretreated with anthracyclines, which makes it a potentially meaningful drug for the treatment of AML."