FDA Rare Pediatric Disease status for AskBio’s AB-1003

German pharma major Bayer’s (BAYN: DE) independently operated subsidiary Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

AskBio noted that the first patient was dosed in Phase I/Phase II LION-CS101 trial of AB-1003 in August 2023, with enrollment continuing.

The FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases that primarily affect children aged 18 years or younger, with fewer than 200,000 people affected in the US.