Dimension Therapeutics Announces FDA Fast Track Designation
Dimension Therapeutics, Inc. recently announced that the US 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 I/II 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 US.
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.
“We are very pleased to achieve this additional important milestone for our DTX101 program, following the FDA’s recent acceptance of Dimension’s investigational new drug (IND) application and granting of orphan drug designation for our lead candidate,” said Annalisa Jenkins, MBBS, MRCP, Dimension’s Chief Executive Officer. “This latest development provides further momentum as we advance DTX101, with the near-term goal to initiate our clinical trial by the end of this year.”
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.
Dimension Therapeutics, Inc. is a rare disease company focused on discovering and developing new therapeutic products for people living with devastating rare diseases associated with the liver and based on the most advanced adeno-associated virus (AAV) delivery technology. The company is advancing multiple programs toward clinical development, including programs addressing unmet needs for patients suffering from OTC deficiency and GSDIa; a collaboration with Bayer HealthCare in hemophilia A, and a wholly owned program in hemophilia B, which is expected to enter clinical testing by the end of 2015. Dimension has preferred access to multiple AAV vectors from REGENXBIO. The Dimension team and senior advisors include biotech industry veterans and renowned thought leaders in gene therapy and rare diseases. For more information, visit www.dimensiontx.com
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