PROVIDENCE – Tivorsan Pharmaceuticals Inc. has received orphan drug designation from the U.S. Food and Drug Administration for a drug used in the treatment of Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness.
The biotechnology company, in a news release this week, said it is working on treating Duchenne muscular dystrophy and other “serious, debilitating neuromuscular disorders.”
“We’re excited to receive orphan drug designation for human recombinant Biglycan and realize this important step toward our goal of bringing a much-needed treatment to patients with DMD,” James E. Connolly, CEO of Tivorsan, said in a statement. “The Tivorsan team is executing on key activities to advance the development of our lead form of Biglycan, which is known as TVN-102.”
He said they expect to start Phase 1 clinical trials next year.
According to the company, this FDA designation provides special status to novel drugs and biologics that diagnose, treat or prevent rare or orphan diseases and disorders that affect fewer than 200,000 patients in the United States.
The designation provides the drug developer with regulatory and financial incentives, including a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, assistance in clinical trial design and regulatory proceedings, along with a waiver of FDA’s drug filing fees.
TVN-102 is based on scientific work from the Fallon Laboratory at Brown University; Justin Fallon, scientific founder of Tivorsan, led the work.
TVN-102 is a small, naturally occurring extracellular matrix protein found in muscle, the release said, adding it has been shown to improve muscle health and function in a mouse model. Mice treated with the drug also show less muscle cell degeneration and more survive.
