“There is an urgent need for new therapeutic options to treat hypoxic-ischemic encephalopathy, a devastating condition that profoundly impacts newborns and their families,” said Ulrich Thienel, M.D., Ph.D., ReAlta’s chief executive officer.
“ReAlta is passionate about supporting this vulnerable population and bringing forward the first pharmacological therapy for the treatment of HIE. The orphan drug designation for our RLS-0071 compound is an important milestone in ReAlta’s strategic plan to advance our PIC1 platform to address life-threatening medical needs.”
The FDA’s Office of Orphan Drug Products grants orphan status to drugs intended to treat rare disorders that affect fewer than 200,000 people in the U.S. The designation provides certain benefits to the drug developer, including seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials.
ReAlta intends to submit an Investigational New Drug (IND) application to FDA for HIE later this year, begin first-in-man clinical studies in late 2020, and then begin HIE Phase 2 studies in neonates in 2021.
Hypoxic Ischemic Encephalopathy (HIE) is also known as neonatal brain asphyxia or ‘blue baby’. In this disease, otherwise normal healthy infants suffer from oxygen starvation to the brain. This can be caused by several processes, including placental tear or tight cord around the neck.
These infants are typically otherwise healthy, and the event is abrupt and unanticipated. Approximately 6,500 to 12,000 newborns in the U.S. are affected every year, or about one every hour. Many of these infants will die after birth or suffer permanent, life-long brain damage, such as severe mental retardation, or cerebral palsy, or both.
HIE is frequently emotionally and financially devastating for the families of HIE babies. Worldwide, HIE is among the top three causes of infant death. The current state-of-the-art treatment for HIE is therapeutic hypothermia; there are currently no pharmacological therapies approved for HIE.
Source: Company Press Release