AML cells
The US Food and Drug Administration (FDA) has granted annamycin orphan designation for the treatment of acute myeloid leukemia (AML).
Annamycin is a liposomal anthracycline under development by Moleculin Biotech, Inc.
The company said it is working with the FDA on an investigative new drug application for a phase 1/2 trial of annamycin in patients with relapsed or refractory AML.
Annamycin has already been tested in 6 clinical trials, 3 of which focused on leukemia.
Results from one of these trials, in adults with relapsed/refractory acute lymphoblastic leukemia, were published in Clinical Lymphoma, Myeloma & Leukemia in 2013.
Annamycin has been under development by several other pharmaceutical companies. Moleculin Biotech, Inc. acquired rights and development assets relating to the drug in 2015.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.
Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.
