SAGE Therapeutics announced that the FDA has granted Orphan Drug designation to SAGE-547 for the treatment of super-refractory status epilepticus (SRSE).
SAGE-547 is an intravenous, allosteric modulator of both synaptic and extra-synaptic GABAA receptors. Numerous drugs targeting GABAA receptors in the CNS have been developed with over decades of research for a variety of CNS disorders. SAGE-547 will be studied as an adjunctive therapy for the treatment of SRSE in an upcoming Phase 1/2 clinical trial.
Status epilepticus (SE) is a life-threatening seizure condition that is initially treated with benzodiazepines. Non-responders to benzodiazepines or second-line anticonvulsants are diagnosed with having refractory SE; these patients require ICU admission and induction into a medically induced coma. If seizure activity continues after anesthetic administration during the coma, the patient is considered to have SRSE, a condition without any approved therapies.
For more information call (617) 299-8379 or visit SAGErx.com.