FDA backs cannabis-based medicine for in-danger newborns
Cambridge biomedical innovator, GW Pharmaceuticals, has won fast track designation from the US FDA for a potential treatment for babies deprived of oxygen during childbirth – a problem that can lead to brain damage and death.
NASDAQ-quoted GW has received FDA support for a proprietary intravenous form of cannabidiol (CBD) to treat Neonatal Hypoxic-Ischemic Encephalopathy, or NHIE.
The European Medicines Agency has granted orphan designation for the same product to treat perinatal asphyxia, an alternate term that describes the same condition as NHIE, for which GW has already received Orphan Drug Designation from the FDA.
There are currently no approved medicines in Europe or the US specifically indicated for NHIE or perinatal asphyxia.
Justin Gover, GW's CEO, said: “GW believes that cannabinoids may have a potentially important role in the treatment of high need paediatric neurologic conditions. As a result, we have developed a proprietary intravenous CBD formulation specifically for use in this most vulnerable of patient populations, newborn infants with NHIE.
“As there are no current treatment options beyond induced hypothermia for the affected newborns, there exists a dramatic need to develop new and effective therapies aimed at preventing acute brain damage and enhancing long-term brain repair.
“GW is now working towards the launch of a clinical development program in this important medical condition towards the end of this year.”
NHIE and perinatal asphyxia are forms of 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 these conditions is estimated to be approximately 12,000 to 24,000 cases per year in the United States and EU combined. Of these cases, 35 per cent of victims are expected to die in early life and 30 per cent may suffer from permanent disability.
The current standard of care for perinatal asphyxia patients is to induce whole-body hypothermia. This treatment is only available in specialised neonatal intensive care units and must be started within six hours of birth. Even if a patient is put into induced hypothermia there is still a significant rate of morbidity and mortality, with a meta-analysis of the available data revealing a 27 per cent death rate.
Among the patients who survive, 28 per cent suffer from major neurodevelopment issues and 26 per cent develop cerebral palsy.
A drug development program with Fast Track designation is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval to get important new drugs to the patient earlier.
GW Pharmaceuticals announced slightly higher revenues but growing losses for the first nine months of 2015 but has been investing massively in R & D. The company has also grown fast at its relatively new operations in California and headcount is already up to 25 there. Its product pipeline has grown enormously in recent months.
The business is also cash rich. Follow-on funding of $193.3 million was raised in May.
Revenue for the nine months ended June 30 were £22.9m compared to £22.6m the previous nine months. Loss for the nine months was up to £32.3m from £14.9m year-on-year. Cash and cash equivalents are £254m – up from £164.5m at September 30.
Gover said: “GW has unprecedented momentum with four phase 3 trials progressing for Epidiolex in paediatric epilepsy as well as a full pipeline of other early and late stage clinical programmes across a range of indications.
“We expect to carry this momentum through the remainder of 2015 as we approach pivotal Phase 3 data for Epidiolex and GW moves confidently forward with preparations for an expected NDA submission in 2016 as well as continuing to build a high quality US commercial infrastructure ahead of commercial launch.”