US FDA Nods paediatric disease and orphan-drug designation to treat LGMD

Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company fully owned and autonomously operating as a subsidiary of Bayer AG, 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).

The FDA grants a rare pediatric disease designation to promote the development of new treatments for serious and life-threatening diseases that primarily affect children 18 years old or younger, with fewer than 200,000 people impacted in the US. If AB-1003 gains approval, AskBio could qualify for a priority review voucher as a result of securing this designation. A priority review voucher can be used for an additional treatment in the company’s pipeline, enabling a shortened review period during the marketing application assessment, or it can be sold and handed over to another company.

Orphan designation provides orphan status to drugs and biologics intended for rare diseases that meet particular criteria, potentially giving a company exclusive marketing privileges for seven years, along with numerous additional benefits.

“These designations for AB-1003 are clear recognition of the significant unmet medical need in LGMD, including type 2I/R9, which is the focus of AskBio’s clinical program and for which there is no approved therapy,” said Canwen Jiang, MD, PhD, chief development officer and chief medical officer, AskBio. “The burden of this rare form of muscular dystrophy on patients and their families is profound, and these decisions support our efforts to potentially bring a new therapeutic option to people living with the 2I/R9 type of this devastating disease.”