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EU tags Abeona's gene therapy as priority medicine

The European Medicines Agency has given priority medicines, or PRIME, status to ABO-102, Abeona Therapeutics Inc.'s gene therapy for treating a genetic metabolic disorder.

New York-based Abeona said the PRIME designation is based on data from an early stage clinical trial called Transpher A for children with Sanfilippo syndrome type A, or mucopolysaccharidosis type IIIA. The condition is a rare enzyme deficiency that mostly affects the central nervous system, causing fatal brain damage.

PRIME designation is provided to medicines that target an unmet medical need. Drug developers are provided with scientific advice and accelerated approval from the European regulator.

ABO-102 was granted an orphan-drug tag by the EMA in October 2016, while the U.S. Food and Drug Administration granted it regenerative medicine status in April 2018.