SOM Biotech, a clinical-stage drug discovery and development company focused on orphan diseases of the Central Nervous System, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SOM3355, currently in clinical development for the treatment of chorea movements in Huntington´s Disease (HD).
The FDA Office of Orphan Products Development grants orphan status to support the development of drugs for the safe and effective treatment of rare disorders. Receiving ODD may qualify SOM Biotech for a seven year period of US marketing exclusivity upon approval of SOM3355.
Raúl Insa, Chief Executive Officer of SOM Biotech highlights: “Receiving Orphan Drug Designation for SOM3355 is a significant step forward for Huntington’s Disease patients and confirms the potential of our AI-powered approach to accelerate drug discovery and development. SOM3355’s profile is a compelling alternative to existing drugs in this space and we are excited by the added momentum that Orphan Drug Designation gives to the project to ensure we can make this treatment available as soon as possible to patients.”
HD is an inherited disorder whereby progressive degeneration of brain centers involved in movement can result in chorea, an involuntary, jerky, movement that randomly affects somatic muscles and flows from one part of the body to another. Using the Company’s proprietary artificial intelligence-based computational technology (SOMAI PRO), SOM Biotech identified SOM3355 or Bevantolol hydrochloride, a drug commonly used to treat hypertension, as a vesicular monoamine transporter 2 (VMAT2) inhibitor that could be repositioned to treat chorea in HD but with a much better side-effect profile than the current standard-of-care.