GW Pharmaceuticals plc has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Cannabidiol (CBD) for use in treating newborn children with neonatal hypoxic-ischemic encephalopathy (NHIE). NHIE is acute or sub-acute brain injury due to asphyxia caused during the birth process and resulting from deprivation of oxygen during birth (hypoxia).
The incidence of NHIE is 1.5 to 2.8 per 1,000 births in the United States, or, 6,500 to 12,000 cases per year. Of these, 35% are expected to die in early life and 30% may suffer from permanent disability. There are currently no FDA-approved medicines specifically indicated for NHIE.
GW has developed an intravenous CBD formulation for use in this patient population. GW held a pre-IND meeting with the FDA and expects to submit an Investigational New Drug Application (IND) in mid-2015 and to commence a Phase 1 trial in the second half of 2015.
GW has been conducting pre-clinical research into the neuroprotective effects of CBD in animal models of NHIE since 2008. Publications from this research have shown that CBD reduces neurologic disability across a range of newborn animal models of brain hypoxia.
"GW continues to explore the opportunity for the development of cannabinoids in rare pediatric conditions characterized by significant unmet need," stated Justin Gover, GW's Chief Executive Officer. "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."
About Orphan Drug Designation
Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition - generally a disease or condition that affects fewer than 200,000 individuals in the U.S. The first NDA applicant to receive FDA approval for a particular active moiety to treat a particular disease with FDA orphan drug designation is entitled to a seven-year exclusive marketing period for that drug and use, unless the applicant is unable to assure sufficient quantities of the drug or another applicant is able to demonstrate that its version of the drug is clinically superior.