Tivorsan receives orphan drug designation for Human Recombinant Biglycan for DMD

Release Date: 20-Oct-2016

Tivorsan pharmaceutical, a company focusing on developing drugs and treatments for all kinds of Duchenne Muscular Dystrophy (DMD) and other neuromuscular disorders, declared that food and Drug Association (FDA) has granted Orphan drug designation to its leading drug candidate, Human Recombinant Biglycan for the treatment of DMD.


Human Recombinant Biglycan, also called as TVN-102, is a small, naturally occurring extracellular matrix protein found in muscle. It has a unique mechanism of action, that of up regulating utrophin, nNOS (neuronal nitric oxide synthase) and other dystrophin-associated proteins in the muscle membrane, as well as controlling MuSK (muscle specific kinase) activity and localization at the nerve-muscle synapse.


FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and 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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