The US Food and Drug Administration (FDA) has granted orphan designation for DTX101 as a treatment for hemophilia B.
DTX101 is designed to deliver factor IX gene expression in a durable fashion to prevent the long-term complications of hemophilia B.
Preclinical studies have indicated that DTX101 has the potential to be a well-tolerated, effective therapy for hemophilia B, according to Dimension Therapeutics, Inc., the company developing DTX101.
The company said it expects to initiate a multicenter, phase 1/2 study to evaluate DTX101 in adults with moderate/severe to severe hemophilia B by the end of this year.
About orphan designation
The FDA grants orphan designation to drugs and biologics intended to treat rare diseases or conditions that affect fewer than 200,000 people in the US.
Orphan designation provides the company developing a drug with certain benefits, such as tax credits for qualified clinical trials, exemption from FDA user fees, and 7 years of market exclusivity if the drug is approved.
