Research Triangle Park, N.C.  – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, 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).

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. If AB-1003 is approved, AskBio may qualify for a priority review voucher based on receipt of this designation. A priority review voucher can be applied to another therapy in the company’s pipeline, enabling a shorter review timeline during marketing application review or can be sold and transferred to another company.

Orphan designation provides orphan status to drugs and biologics for rare diseases that meet certain criteria and potentially gives a company exclusive marketing rights for a seven-year period, along with other 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.”

LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.People living with LGMD2I/R9 may notice symptoms including loss of mobility, impaired heart or lung function. These symptoms can occur in school age and younger children. As symptoms worsen, individuals generally require wheelchairs. LGMD2I/R9 is a rare disease, estimated to affect fewer than 5,000 people in the US. Currently, there is no treatment that modifies disease progression, and management is based on the signs and symptoms present in each individual.

With a broad portfolio of investigational gene therapies at various stages of research and development, AskBio continues to develop adeno-associated virus (AAV)-based therapies to treat some of the world’s most debilitating diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications and aims to deliver breakthrough treatments that could potentially benefit tens of millions of patients worldwide.