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Orphan Drug Approved for Huntington’s Disease
Omeros Corporation has announced that OMS824, its phosphodiesterase 10 (PDE10) inhibitor, has received an orphan drug designation from the FDA for the treatment of patients with Huntington’s disease. An orphan designation is granted to promote the development of drugs targeting conditions in 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments.
This investigational agent selectively inhibits PDE10, an enzyme expressed in areas of the brain and linked to various diseases that affect cognition, such as Huntington's disease and schizophrenia.
In a Phase I study, the drug was well tolerated in healthy subjects and appeared to have a better clinical therapeutic index (safety factor) than other PDE10 inhibitors in development. The company plans to begin a Phase II clinical trial to evaluate OMS824 in patients with Huntington's disease later this year. A Phase II clinical trial of the drug is already under way in patients with schizophrenia.
It is estimated that Huntington’s disease affects approximately 31,000 U.S. patients each year. The only FDA-approved treatment so far is Tetrabenazine (Xenazine, Valeant/Lundbeck), which is indicated for Huntington’s-related movement disorders. OMS824 has the potential to improve the cognitive and psychiatric abnormalities as well as the movement disorders associated with the disease.[Source: Omeros Corp., September 30, 2013.]