EMA announces orphan drug designation for ABO-102 gene therapy

Release Date: 09-Nov-2016

Abeona Therapeutics Inc., a biopharmaceutical company dedicated to develop gene therapies for life threatening rare diseases declared that leading therapy ABO-102 has been designated as orphan drug b European Medicines Agency (EMA) for the treatment of Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease causing neurocognitive decline, speech of loss, premature death in children and loss of mobility.


Abeona’s MPS IIIA program has been previously granted the designation of orphan drug by Food and Drug Association (FDA) in the US and received the Rare Pediatric Disease Designation as a pre-requisite part of the Priority Review Voucher (PRV) process.


The FDA and EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan Status can be applied to products proven safe and effective in treating conditions that affect a relatively small number of patients, and the designation may entitle up to seven years of US marketing exclusivity upon regulatory approval. Companies whose drugs have an orphan designation also receive certain tax credits and are exempted from paying prescription drug user fees normally required of companies submitting products for approval.



For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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