EU approval for Abeona’s gene therapy to treat Sanfilippo Syndrome Type A

19 October 2016  •  Author: Niamh Louise Marriott, Digital Content Producer

The European Medicines Agency (EMA) Committee has granted orphan drug designation for Abeona‘s lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.


Abeona’s MPS IIIA program, ABO-102, has previously been granted FDA orphan product designation in the US and received the rare paediatric disease designation as a pre-requisite part of the priority review voucher (PRV) process.

“Receiving European Union orphan drug designation is an important milestone that delivers significant commercial benefits to our company,” stated Timothy Miller PhD President & CEO of Abeona Therapeutics Inc. “The benefits and incentives associated with these designations, including marketing exclusivity, are strategically important from a regulatory and commercial perspective and potentially value-creating for shareholders.”

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