FDA Grants Encoded RMAT Designation for SCN1A+ Dravet Syndrome

Rare Daily Staff

The U.S. Food and Drug Administration has granted Encoded Therapeutics Regenerative Medicine Advanced Therapy designation for its experimental therapy ETX101 for the treatment of the rare developmental epilepsy SCN1A+ Dravet syndrome.

Regenerative Medicine Advanced Therapy, or RMAT, is an expedited program to facilitate the development and review of regenerative medicine therapies that are intended to treat, modify, reverse, or cure a serious or life-threatening disease. The RMAT designation provides Encoded with early, close, and frequent interactions with the FDA; organizational commitments from senior managers; and other opportunities to expedite the review of ETX101.

Dravet syndrome is a severe, lifelong disorder of the central nervous system that occurs in about one in 16,000 births worldwide. Most cases result from loss-of-function variants in the SCN1A gene. This developmental and epileptic encephalopathy affects people of both sexes and all races, manifesting in a wide array of symptoms. Frequent, prolonged, and drug-resistant seizures typically begin in the first year of life in an otherwise developing infant. Severe cognitive and developmental stagnation, sleep abnormalities, motor impairment, and behavioral challenges usually become clinically evident by the second or third year of a child’s life.

ETX101 is an AAV9-based gene regulation therapy designed to increase SCN1A expression in GABAergic inhibitory neurons for the treatment of SCN1A+ Dravet syndrome. ETX101 has Fast Track, Rare Pediatric, and Orphan Drug designations from the FDA, as well as an Orphan Designation from the European Medicines Agency.

“The FDA’s decision to grant RMAT designation highlights the promising clinical efficacy and safety profile we have observed to date,” said Kartik Ramamoorthi, CEO of Encoded Therapeutics. “This milestone, together with the transition to in-house GMP manufacturing, underscores both the clinical potential of ETX101 and our readiness to efficiently advance the program through development.”

Photo: Kartik Ramamoorthi, CEO of Encoded Therapeutics