Praxis Precision Medicines Inc.
(NASDAQ:PRAX) said on Monday that the U.S.
Food and Drug Administration granted Breakthrough Therapy Designation to elsunersen (PRAX-222), its investigational therapy for seizures associated with SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) caused by gain-of-function variants in the SCN2A gene.
SCN2A-DEE is a rare genetic epilepsy that typically begins during infancy and is associated with severe developmental and neurological impairments.
The designation marks the third Breakthrough Therapy Designation for the company's late-stage pipeline and is expected to accelerate the development and regulatory review process for the experimental treatment.
