SAGE Therapeutics (SAGE), a biopharmaceutical company developing novel
medicines to treat life-threatening, rare central nervous system (CNS)
disorders, today announced the U.S. Food and Drug Administration (FDA)
has granted orphan drug designation to its neuroactive steroid,
SAGE-547, for the treatment of status epilepticus. SAGE Therapeutics is
currently evaluating SAGE-547 in a Phase 1/2 clinical trial for the
treatment of super-refractory status epilepticus (SRSE).
"Receiving orphan drug designation may help SAGE with the development of
SAGE-547 and may ultimately allow us to deliver this promising medicine
to patients who suffer from status epilepticus," said Jeff Jonas, M.D.,
chief executive officer of SAGE Therapeutics. "Status epilepticus is a
life-threatening disease with limited treatment options, and this
advancement demonstrates our commitment to delivering SAGE-547 and other
therapies to patients with life-threatening, rare CNS disorders."
Orphan drug designation, which is intended to facilitate drug
development for rare diseases, provides substantial benefits to the
sponsor, including the potential for tax credits for clinical
development costs, study-design assistance, and several years of market
exclusivity for the product upon regulatory approval.
SAGE-547 is an allosteric modulator of both synaptic and extra-synaptic
GABAA receptors. GABA receptors are widely
regarded as validated drug targets for a variety of CNS disorders, with
decades of research and multiple approved drugs targeting these receptor
systems. SAGE-547, developed by SAGE Therapeutics using its proprietary
chemistry platform, is an intravenous agent in Phase 1/2 clinical
development as an adjunctive therapy, a therapy combined with current
therapeutic approaches, for the treatment of SRSE.
About Status Epilepticus
SE is a life-threatening seizure condition that occurs in approximately
150,000 people each year in the U.S.,1 of which 30,000 SE
patients die. We estimate that there are 35,000 patients with SE in the
U.S. that are hospitalized in the intensive care unit (ICU) each year.
An SE patient is first treated with benzodiazepines, and if no response
then treated with other, second-line, anti-seizure drugs. If the seizure
persists after the second-line therapy the patient is diagnosed as
having refractory SE (RSE), admitted to the ICU and placed into a
medically induced coma. Currently, there are no therapies that have been
specifically approved for RSE; however, physicians typically use
anesthetic agents to induce the coma and stop the seizure immediately.
After a period of 24 hours, an attempt is made to wean the patient from
the anesthetic agents to evaluate whether or not the seizure condition
has resolved. Unfortunately, not all patients respond to weaning
attempts, in which case the patient must be maintained in the medically
induced coma. At this point, the patient is diagnosed as having SRSE.
Currently, there are no approved therapies for SRSE.
About SAGE Therapeutics
SAGE Therapeutics is a biopharmaceutical company committed to developing
and commercializing novel medicines to treat life-threatening, rare CNS
disorders. SAGE's lead program, SAGE-547, is in clinical development for
SRSE and is the first of several compounds the company is developing in
its portfolio of potential seizure medicines. SAGE's robust chemistry
platform has generated multiple new compounds that target the GABAA
and NMDA receptors, which are broadly accepted as impacting many
psychiatric and neurological disorders. SAGE Therapeutics is a private
company launched in 2010 by an experienced team of R&D leaders, CNS
experts and investors. For more information, please visit www.sagerx.com.
1 DeLorenzo, Robert J., Pellock, John M., Towne, Alan R.,
Boggs, Jane G. Epidemiology of Status Epilepticus. J Clin Neuro
1995; 12(4): 316-325.
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