Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company and subsidiary of Bayer AG, has received rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
The FDA grants rare pediatric disease designation to encourage the development of new therapies for serious and life-threatening diseases primarily affecting children aged 18 years or younger, with fewer than 200,000 affected individuals in the U.S. If AB-1003 gains approval, AskBio may be eligible for a priority review voucher, potentially expediting the review timeline for another therapy in their pipeline or for sale to another company.
Orphan drug designation provides drugs and biologics for rare diseases that meet specific criteria with potential marketing rights for a seven-year period, along with other benefits.
Addressing Unmet Needs in LGMD2I/R9
"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."
Understanding LGMD2I/R9
LGMD2I/R9 is a form of LGMD caused by mutations in the FKRP gene, leading to weakness and wasting of arm and leg muscles. Symptoms, including loss of mobility and impaired heart or lung function, can manifest in school-aged children and younger. As the disease progresses, individuals often require wheelchairs. LGMD2I/R9 is a rare condition, affecting an estimated fewer than 5,000 individuals in the U.S. Currently, management focuses on addressing individual signs and symptoms, as there are no treatments to modify disease progression.
AskBio's Gene Therapy Approach
AskBio is advancing a broad portfolio of investigational gene therapies using adeno-associated virus (AAV)-based technology to treat debilitating diseases. Their clinical programs span neuromuscular, central nervous system, cardiovascular, and metabolic disease indications, aiming to deliver breakthrough treatments for millions of patients worldwide. The company's clinical-stage pipeline includes investigational therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.
