Note: This is an edited form of a press release from Dimension Therapeutics, Inc. To read the original release in its entirety, click here.
Dimension Therapeutics, Inc. announced on Thursday, September 17 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s lead product candidate, DTX101, for the treatment of hemophilia B. Dimension expects to initiate a multi-center phase 1/2 study to evaluate DTX101 in adult patients with moderate/severe to severe hemophilia B by the end of 2015.
DTX101 is designed to deliver blood clotting Factor IX (FIX) gene expression in patients with hemophilia B. Hemophilia B is a rare genetic bleeding disorder resulting from a deficiency in FIX. The current standard of care for patients with hemophilia B involves chronic replacement of FIX protein through intravenous infusion. In 2013, the World Federation of Hemophilia estimated there were more than 28,000 hemophilia B patients worldwide, including 4,000 patients in the United States.
The FDA’s Fast Track program is designed to facilitate and expedite development and review of new drugs to treat serious or life-threatening conditions and address unmet medical need. Through the Fast Track program, a product may be eligible for priority review at the time of BLA and may be eligible to submit sections of the BLA on a rolling basis as data become available. In addition there are opportunities for frequent interactions with FDA with this designation.
Dimension is developing its lead gene therapy product DTX101, which is expected to enter clinical development in 2015, for the treatment of moderate/severe to severe hemophilia B. DTX101 is designed to deliver Factor IX, or FIX, gene expression in a durable fashion, preventing the long-term complications of hemophilia B. Preclinical studies completed to date indicate DTX101 has the potential to be a well-tolerated, effective therapy for hemophilia B.