Abeona receives orphan drug designation for ABO-101 to treat Sanfilippo Syndrome Type- B

Release Date: 14-Feb-2017

Abeona therapeutics, a clinical stage bio-pharmaceutical company dedicated to develop gene therapies for life threatening diseases, has recently announced that its novel gene therapy candidate, ABO-101 has been awarded with orphan drug status for the treatment of children with Sanfilippo Syndrome Type-B, by the European Medicine Agency (EMA), a committee for orphan medicinal products. Sanfilippo Syndrome Type-B is a rare genetic disease causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

Previously, the product has been grant with orphan drug designation by US Food and Drug Administration (FDA). The organization has allowed the drug for clinical phase andfrac12; trial and enrollments are due in the second quarter of 2017.

The European Commission grants orphan drug designation status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including protocol assistance, reduced fees and protection from market competition once ABO-101 is approved for the treatment of MPS IIIB patients.

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