The U.S. Food and Drug Administration (FDA) today approved SD-809 (deutetrabenazine), the second drug approved for use in the United States to treat chorea in Huntington disease (HD), a rare, inherited neurodegenerative disorder.
The approval was based on positive results from the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Group (HSG) on behalf of Teva Pharmaceuticals. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience. The results were published in JAMA, July 2016.
“We are so grateful to the patients and families who made this development possible by participating in this ground-breaking trial. Trial participants are the key to bringing new treatments to the entire HD community,” said Samuel Frank, MD, Huntington Study Group’s principal investigator for First-HD and associate professor of Neurology at Beth Israel Deaconess Medical Center/Harvard Medical School. Claudia Testa, MD, PhD, associate professor of Neurology at Virginia Commonwealth University served as the co-principal investigator.
“It’s exciting to offer a new treatment,” Testa said. “Trials like these give patients, families, and care providers more options to effectively manage HD symptoms and improve quality of life.”
Most individuals with HD experience chorea during the course of the disease. Huntington disease is an autosomal-dominant, inherited disease that usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others experience disease symptoms much later in life. The disease brings with it an array of symptoms besides chorea, including dystonia, cognitive problems, changes in personality, and psychiatric problems like depression. Because HD is autosomal dominant, each child of a person with HD has a 50 percent chance of inheriting the genetic change that causes the disease from their affected parent, whether that parent is their mother or father. For more information about HD, visit www.huntingtonstudygroup.org.
Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. Deutetrabenazine is the first FDA approved compound with deuterium substitution. Much of the clinical work that led to the approval of deutetrabenazine was carried out by the Huntington Study Group, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, and Australia who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information, visit www.huntingtonstudygroup.org.
“This is a great day for the HD community,” said Ray Dorsey, MD, chair of the Huntington Study Group and director of the University of Rochester’s Center for Human Experimental Therapeutics (CHET). “The unmet need for therapeutics for individuals with HD is immense, and this approval brings us closer to making HD an increasingly treatable condition.”
First-HD was conducted at 34 Huntington Study Group sites across the United States and Canada, enrolling 90 participants over 14 months, in the 13-week double-blind, placebo-controlled trial. Scientific, technical, logistical, and analytical support for the study was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders. For more information about the Clinical Trials Coordination Center, visit www.ctcc.rochester.edu.
Teva Pharmaceuticals owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals in 2015. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.
A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This open-label trial is also being led by the Huntington Study Group and the Clinical Trials Coordination Center for Teva Pharmaceuticals. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.
Huntington Study Group