ABX1100 Given Rare Pediatric Drug Designation by FDA


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.


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

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