Posted by Michael Wonder on 26 Oct 2016
Abeona Therapeutics announces fast track designation from FDA for ABO-102 in Sanfilippo syndrome type A
25 October 2016 - Fast track designation added to already granted orphan drug designation by the FDA and EMA, and rare paediatric disease designation for ABO-102 in Sanfilippo syndrome type A.
Abeona Therapeutics announced today that the U.S. FDA granted fast track designation for ABO-102, a single intravenous injection of AAV gene therapy for subjects with mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.
