FDA announces orphan drug designation for DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa)

Release Date: 15-Nov-2016

Dimension Therapeutics Inc., a biopharmaceutical company specialized in developing gene therapies targeting liver related diseases, declared that US Food and Drug Association (FDA) granted orphan drug status for DTX401 for the treatment of patients with Glycogen Storage Disease Type Ia (GSDIa). DTX401 is the first AAV gene therapy to enter IND-enabling studies for the treatment of GSDIa.   



DTX401 has been designed to serve underlying genetic effect for which it delivers stable expression and activity of the enzyme glucose-6-phosphatase (G6Pase). It improves G6Pase activity and reduces hepatic glycogen levels, a well-described biomarker of disease progression, in multiple in vivo preclinical models.



FDA provides a number of benefits to the drug developers or pharmaceutical companies through development and commercialization. Orphan Drug designation is granted by the FDA's Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.


For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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