Saturday, May 09, 2015

Cannabidiol Given Orphan Drug Designation for NHIE by the FDA

The drug Cannabidiol was recently granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of children with neonatal hypoxic-ischemic encephalopathy (NHIE).1
Neonatal deprivation of oxygen during the birth process can lead to the acute or sub-acute brain injury known as NHIE.1 In the US, the incidence of NHIE is 1.5 to 2.8 per 1,000 births.1   Inflammatory cytokines, oxidative stress, and cell death all play a role in the disease pathology.2 Affected infants may show loss muscle tone, seizures, poor feeding, and a depressed level of consciousness.2  Around 35% of these infants to die in early life, and 30% may suffer from permanent disability.1  Currently, there are no effective FDA-approved medications to treat the condition.1
Cannabidiol is a non-psychoactive ingredient in cannabis. It is one of a group of compounds, the cannabinoids, which mediate their effects through the cannabinoid receptors. 3 Several studies have highlighted the potential of cannabinoids as neuroprotective treatments in ischemic brain damage, perhaps partly because of their anti-inflammatory and antioxidant properties.4
Since 2008, GW has been researching the neuroprotective effects of Cannabidiol in animal models of NHIE. This work has shown that Cannabidiol reduces neurologic disability in several models of newborn animal brain hypoxia. GW expects to submit an Investigational New Drug Application (IND) in mid-2015 for their intravenous formulation of CBD. The company expects to begin a Phase 1 trial in the second half of this year.1

"This orphan drug designation for Cannabidiol for the treatment of newborn children with neonatal hypoxic-ischemic encephalopathy follows several years of pre-clinical evaluation," GW’s Chief Executive Officer, Justin Gover, stated in a press release.1 Potentially, Cannabidiol might be approved for orphan drug status for other uncommon medical conditions, especially those with an inflammatory component.3 Mr. Grover also noted, “GW continues to explore the opportunity for the development of cannabinoids in rare pediatric conditions characterized by significant unmet need.”1


  1. GW Pharmaceuticals receives orphan drug designation from FDA for Cannabidiol for the treatment of neonatal hypoxic-ischemic encephalopathy [press release]. Published April 24, 2015. Accessed May 3, 2015.
  2. Fatemi A, Wilson MA, Johnston MV. Hypoxic ischemia encephalopathy in the term infant. Clin Perinatol. 2009;36(4):834-838. Accessed May 3, 2015.
  3. Nagarkatti P, Pandey R, Rieder SA, Hegde V, Nagarkatti M. Cannabinoids as novel anti-inflammatory drugs. Future Med Chem. 2009;1(7):1333-1349. Accessed May 3, 2015.
  4. Alvarez FJ, Lafuente H, Rey-Santano MC, et al. Neuroprotective effects of the nonpsychoactive cannabinoid cannabidiol in hypoxic-ischemic newborn piglets. Pediatr Res. 2008;64:653-658. Accessed May 3, 2015.