Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Abeona Therapeutics’ ABO-101, an experimental gene therapy for Sanfilippo syndrome type B (MPS IIIB), a rare and deadly lysosomal storage disorder.

MPS IIIB is caused by a build-up of toxic sugars called glycosaminoglycans (GAGs) due to a mutation in the gene encoding the NAGLU enzyme. Symptoms include rapid neurodevelopmental and physical decline, aggressive behavior, seizures, loss of speech or vision and premature death. An estimated 70 percent of children with MPS III do not reach the age of 18.

ABO-101 is designed to deliver a normal copy of the NAGLU gene and prevent abnormal build-up of GAGs via one-time intravenous administration. The therapy is designed to address the underlying lysosomal enzyme deficiency responsible for abnormal accumulation of GAGs in the brain and throughout the body that result in progressive cell damage and neurodevelopmental and physical decline. Abeona is enrolling eligible patients with MPS IIIB at sites in the United States and Spain and expects to activate additional sites globally this year to accelerate enrollment.

“Receiving Fast Track designation is recognition by FDA that ABO-101 shows promise in treating a serious life-threatening disease and may address the significant unmet medical need of children with MPS IIIB,” said João Siffert, CEO of Abeona. “We will continue to interact with global regulatory authorities about the ABO-101 clinical program to facilitate development of this one-time AAV9 mediated gene therapy.”

Fast Track designation is granted by the FDA to facilitate the development and expedite review of investigational therapies intended to treat serious or life-threatening diseases that show potential for addressing significant unmet medical needs. Clinical development of drug products receiving the designation benefit from frequent FDA interactions during all aspects of development and if specific criteria are met, they are eligible for Priority Review and Accelerated Approval. ABO-101 has also received Orphan Drug designations in the United States and European Union, and Rare Pediatric Disease designation from the FDA.

Photo: João Siffert, CEO of Abeona