Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned by Bayer AG, announced that AB-1003 (LION-101) has received rare pediatric disease and orphan-drug designations from the FDA for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
The FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases primarily affecting 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, potentially accelerating the review timeline for another therapy in their pipeline.
Orphan-drug designation provides drugs and biologics for rare diseases that meet certain criteria with orphan status, potentially giving a company exclusive 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. She emphasized the profound burden of this rare form of muscular dystrophy on patients and their families, noting that these designations support their efforts to potentially bring a new therapeutic option to people living with the 2I/R9 type of this devastating disease.
LGMD2I/R9 is caused by changes 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-age children and younger. As the disease progresses, individuals often require wheelchairs. Affecting fewer than 5,000 people in the US, LGMD2I/R9 currently lacks disease-modifying treatments, with management focused on addressing individual signs and symptoms.
AskBio's Gene Therapy Approach
AskBio is advancing a broad portfolio of investigational gene therapies using adeno-associated virus (AAV)-based therapies to treat debilitating diseases. Their clinical programs span neuromuscular, central nervous system, cardiovascular, and metabolic disease indications, aiming to deliver breakthrough treatments to millions of patients worldwide.
About Limb-Girdle Muscular Dystrophy
Limb-girdle muscular dystrophy (LGMD) encompasses a group of diseases characterized by progressive weakness and wasting of the muscles in the arms and legs, particularly those closest to the body (proximal muscles). The severity, age of onset, and specific features vary among subtypes and can be inconsistent. Symptoms may appear at any age and generally worsen over time, although some cases remain mild. The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.
