Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company fully owned and operated as a subsidiary of Bayer AG, announced that its investigational gene therapy, AB-1003 (also known as LION-101), has been granted both Rare Pediatric Disease and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) for the treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).
The FDA’s Rare Pediatric Disease designation is awarded to therapies for serious or life-threatening conditions that primarily affect children under 18 and have fewer than 200,000 affected individuals in the U.S. This designation may allow AskBio to qualify for a Priority Review Voucher (PRV) if AB-1003 is approved. The PRV could expedite the review of another drug in the company’s pipeline or potentially be sold to another company for use with a different application.
The Orphan Drug designation is granted to therapies for rare diseases, offering a range of benefits, including potential seven years of exclusive marketing rights upon FDA approval, along with other regulatory advantages.
“These FDA designations for AB-1003 underscore the significant unmet medical need in LGMD, including the specific 2I/R9 subtype, for which there are no approved treatments,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer of AskBio. “The impact of LGMD2I/R9 on affected individuals and their families is profound, and these designations support our commitment to developing a new potential therapy for this devastating disease.
