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Askbio Receives US FDA Rare Paediatric Disease & Orphan-Drug Designations for AB-1003

11 Nov 2024
Admin
News Article

Askbio Receives US FDA Rare Paediatric Disease & Orphan-Drug Designations for AB-1003

AskBio receives US FDA rare paediatric disease & orphan-drug designations for AB-1003 to treat limb-girdle muscular dystrophy type 2I/R9

Overview

Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that AB-1003 (also known as LION-101) has received rare paediatric 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

FDA grants rare paediatric 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

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.

Words from the CDO: AskBio

“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.”

About LGMD2I/R9

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.

AAV-Based Therapies by AskBio

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.

Limb-Girdle Muscular Dystrophy

Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and legs.
The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.
The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are often inconsistent, even within the same family.
Signs and symptoms may first appear at any age and generally worsen with time, although in some cases they remain mild.
Visit the National Institutes of Health Medline Plus website to learn more about LGMD.

About Asklepios BioPharmaceutical, Inc.

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives.
The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.