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# Orphan Drug Status for Tivorsan Duchenne Drug
- URL: https://www.fdaweb.com/orphan-drug-status-for-tivorsan-duchenne-drug/
- Published: 2016-08-15T12:00:00.000Z
- Updated: 2026-09-14T21:24:32.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5136500

FDA has granted Tivorsan Pharmaceuticals an orphan drug designation for TVN-102 (biglycan) and its use in treating Duchenne Muscular Dystrophy. The company says it is on track to submit an IND and begin clinical trials in 2017.

TVN-102 is described by Tivorsan as a small, naturally occurring extracellular matrix protein found in muscle. “It has a unique mechanism of action, that of upregulating 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,” it says. “Tests in the most widely used mouse model of DMD have shown that systemically delivered TVN-102 upregulates utrophin and activates the dystrophin-independent membrane stabilization program. TVN-102 has been shown to improve muscle health and function in a mouse model. Mice treated with TVN-102 also show reduced muscle cell degeneration and death.”