FDA Grants Fast-Track Designation to New Hypoxic-Ischemic Encephalopathy Treatment

Last year, the FDA (Food and Drug Administration) announced that it would provide a ‘fast track’ designation to GW Pharmaceuticals’ intravenous cannabidiol treatment for hypoxic-ischemic encephalopathy, or HIE. This designation is granted to speed the development of products that treat serious or life-threatening conditions when those products show that they can address unmet medical needs. The end goal of this designation is getting important new treatments to patients more quickly by allowing companies to expedite FDA review of their products and improve the efficiency of development.

Clinical trials are set to commence in the second half of this year.

Why Is Fast-Tracking of HIE Treatment Significant?

The fast-tracked status of this new cannabidiol treatment for newborns with HIE (also known as perinatal asphyxia, intrapartum asphyxia or neonatal encephalopathy) highlights the urgent need to find more and better ways of reducing infants’ disability after they suffer oxygen deprivation at birth. Currently, there is only a single treatment for HIE  – hypothermia therapy, otherwise known as brain cooling – and it must be administered within six hours of birth for it to be effective. It is only available in specialized neonatal ICUs, which can limit its viability as a treatment in hospitals that do not have such specialized services.

Cannabidiol Treatment for Hypoxic-Ischemic Encephalopathy Granted Orphan Status in Europe and the U.S.

Both the European Medicines Agency and the FDA have granted GW this cannabidiol treatment ‘orphan drug’ status, a designation reserved for new drugs and biological substances intended for safe and effective treatment, diagnosis and prevention of rare diseases and disorders that affect fewer than 200,000 people (in the U.S.) or fewer than 5 in 10,000 (in the E.U.)

Hypoxic-Ischemic Encephalopathy Affects a Small But Sizable Segment of the U.S. Population

HIE (birth asphyxia) occurs in between 1.4 and 2.8 births per 1,000 live births in the U.S. More than ⅓ (35%) of these children die in early life while an additional 30% suffer from a permanent disability. Given the severity of oxygen deprivation impact on newborns, it is imperative that research into new treatments be expedited to allow babies to have a better quality of life and reduced disability.

“A Dramatic Need to Develop New and Effective Therapies”

According to  Justin Gover, CEO of GW Pharmaceuticals, cannabinoids “may have a potentially important role in the treatment of high need pediatric neurologic conditions.” Preclinical research demonstrates that cannabidiol reduces neurologic disability in numerous newborn animal models of brain hypoxia, providing hope that similar results would be found in the human neonatal population.

According to Global News Wire, Phase 1 clinical trials are expected to begin in the 2nd half of the 2016 fiscal year.


Brain research is an ongoing process, and, at the moment, hypothermia therapy is the standard of care when it comes to hypoxic-ischemic encephalopathy. Additive or supplemental measures in addition to hypothermia are still in preclinical or clinical trials, so it is too early to say whether any of these supplemental measures will be adopted as standards of care. Because these are highly experimental findings, we caution readers that we are merely providing this information as an update on findings in the field of HIE research, rather than as a potential source of treatment information. 

Legal Help for Hypoxic Ischemic Encephalopathy (HIE) Treatment

If your child has disabilities or developmental delays, and you are concerned that this may have been due to a medical mistake, feel free to reach out to the birth injury attorneys at Reiter & Walsh ABC Law Centers. Our unique focus on solely birth injuries allows us to take on these complex cases and secure your child’s future.

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