FDA Grants Orphan Drug Designation To Omeros' OMS824 For Huntington's Disease

(RTTNews) - Omeros Corp. (OMER) said that OMS824, its phosphodiesterase 10 (PDE10) inhibitor, has received orphan drug designation from the U.S. Food and Drug Administration for Huntington's disease treatment.

OMS824 selectively inhibits PDE10, an enzyme expressed in areas of the brain linked to a wide range of diseases that affect cognition, including Huntington's disease and schizophrenia. As reported earlier, Phase 1 results in healthy subjects showed that OMS824 is well tolerated and suggest that it has a better clinical therapeutic index or "safety factor" than other PDE10 inhibitors in development.

Omeros may begin a Phase 2 study, evaluating OMS824 in patients with Huntington's disease later this year, while a Phase 2 trial of the drug is already underway in schizophrenia patients.

Huntington's disease is estimated to affect about 31,000 U.S. patients annually, and the only FDA-approved treatment for the disease is tetrabenazine, which is indicated for Huntington's-related movement disorders. OMS824