On February 21, 2013, the U.S. Food and Drug Administration (“FDA”) granted orphan drug status to an aplastic anemia treatment. Aplastic anemia is a rare condition where bone marrow does not produce sufficient blood cells or platelets. The treatment, placental expanded (PLX) cells, is sponsored by Pluristem Therapeutics, Inc. You can read more about the requirements for obtaining orphan drug status here.
Pursuant to the Orphan Drug Designation program, the FDA provides incentives for the development of products that will diagnose or treat rare diseases. Orphan drug status qualifies a drug sponsor to receive tax credits for clinical testing of the product and other marketing benefits. Otherwise, because it is intended to treat a rare disease, the development of the drug might not be profitable and such rare diseases may never be treated.
To qualify for orphan drug status under FDA regulations, the drug or biologic product must be intended to treat or diagnose a rare disease or condition affecting fewer than 200,000 people per year in the United States. 21 C.F.R. part 316. Additionally, an application must be submitted by the sponsor, and thereafter approved by, the FDA Office of Orphan Products Development.
If you have any questions about the Orphan Drug Designation program or FDA’s regulations of drugs, please contact us at firstname.lastname@example.org.