AskBio’s AB-1003 Receives Rare Pediatric Disease and Orphan Drug Designations from FDA

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned subsidiary of Bayer AG (ETR: BAYN), has announced that it has received both rare pediatric disease designation (RPDD) and orphan-drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational gene therapy, AB-1003 (LION-101). The therapy targets limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a rare and severe genetic disorder.

AB-1003’s Mechanism and FDA Recognition
AB-1003 is an investigational AAV gene therapy that delivers the non-mutated FKRP gene into the body through a single intravenous infusion, aiming to treat the LGMD2I/R9 disease. This approach has been recognized by the FDA with both RPDD and ODD, highlighting the therapy’s potential to address an unmet medical need for a pediatric population with a rare disease. The drug has also been previously awarded fast-track status in the US, underscoring its significance in expediting the development and review process.

Phase I/II Study and Assessment
AB-1003 is currently being assessed in a Phase I/II study in the US, focusing on safety, tolerability, and efficacy. The study’s results will be crucial in determining the therapy’s potential role in improving outcomes for patients with LGMD2I/R9, a condition that currently lacks effective treatment options.-Fineline Info & Tech