FDA grants orphan drug status to cannabidiol for neonatal hypoxic/ischemic encephalopathy

The FDA recently granted orphan drug designation to a pharmaceutical cannabidiol for the treatment of newborn children with neonatal hypoxic/ischemic encephalopathy, according to a press release from the drug’s manufacturer.

An acute or sub-acute brain injury due to asphyxia during the birthing process, neonatal hypoxic/ischemic encephalopathy occurs in 1.5 to 2.8 per 1,000 births in the United States — of these children, 35% are estimated to die in early life while 30% could suffer from permanent disability.

Currently, there are no approved therapies for neonatal hypoxic/ischemic encephalopathy.

Based on preclinical research which demonstrated that cannabidiol reduces neurologic disability across a range of newborn animal models of brain hypoxia, an intravenous formulation of cannabidiol (GW Pharmaceuticals) was developed for use in the neonatal population.

“GW continues to explore the opportunity for the development of cannabinoids in rare pediatric conditions characterized by significant unmet need,” Justin Gover, CEO of GW Pharmaceuticals, said in a press release. “This orphan drug designation for cannabidiol for the treatment of newborn children with neonatal hypoxic-ischemic encephalopathy follows several years of pre-clinical evaluation and we look forward to advancing a clinical development program in this important medical condition later this year.”

GW Pharmaceuticals recently held a pre-Investigational New Drug meeting with the FDA and is expected to submit an Investigational New Drug Application in mid-2015, followed by the launch of a phase 1 trial in the second half of 2015.

The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.