Aro Biotherapeutics’ ABX1100, used to treat Pompe disease, was granted a Rare Pediatric Drug designation by the FDA.
The US Food and Drug Administration (FDA) has granted ABX1100(Aro Biotherapeutics) a Rare Pediatric Drug (RPD) designation.
ABX1100 is an investigational Centyrin-siRNA conjugate used to treat Pompe disease by targeting the Gys1 gene. This reduces glycogen buildup in the muscles, making ABX1100 a novel treatment.
Pompe disease is a rare but often fatal disease, capable of manifesting as early as infancy. A mutation in the acid alpha glucosidase enzyme causes glycogen to break down in muscle cells, weakening and debilitating muscles over time.
“Pompe disease affects patients of all ages but can be particularly devastating in children,” said Mittie Doyle, MD, FACR, chief medical officer of Aro Biotherapeutics. “We are committed to developing ABX1100 for children that suffer from Pompe disease in order to provide these pediatric patients with a new therapeutic option.”
RPD designation is given to drugs which treat serious and life-threatening diseases in pediatric patients. A Pediatric Review Voucher is offered to companies granted an RPD by the FDA, giving them priority review, reduced review time, and potential expedited product approval.
Aro Biotherapeutics is dedicated to developing genetic medications which target tissues using Centyrins, a proprietary protein technology. They are working alongside industry partners to develop therapeutics using Centyrins to target tissues for the treatment of a wide variety of diseases.
“Obtaining RPD designation from the FDA represents an important step for Aro towards our goal of developing novel, tissue targeted genetic medicines for patients with rare diseases, and we are very pleased by the FDA’s recognition of the potential of ABX1100 in the pediatric Pompe community,” said Sue Dillon, PhD, cofounder and CEO of Aro Biotherapeutics.
This article originally appeared on Contemporary Pediatrics.
Reference
Aro biotherapeutics receives FDA rare pediatric drug designation for ABX1100 for the treatment of Pompe disease. Business Wire. September 15, 2022. Accessed September 16, 2022. https://www.businesswire.com/news/home/20220915005101/en/Aro-Biotherapeutics-Receives-FDA-Rare-Pediatric-Drug-Designation-for-ABX1100-for-the-Treatment-of-Pompe-Disease
Continuous Glucose Monitoring Could Improve Outcomes in Pediatric Patients With Leukemia/Lymphoma
July 2nd 2024A pilot study presented at the American Diabetes Association 84th Scientific Sessions showed that CGM technology could produce reliable results without complications in children with leukemia/lymphoma at risk for hyperglycemia.
Socioeconomic Disparities Persisted Alongside Improving Vaccination Rates In US Over 11-Year Period
April 16th 2024Findings indicating that socioeconomic disparities widened gaps in vaccination timeliness signal the need for increased efforts to promote timely vaccination among children from families with lower income and those without private insurance.