RARE Daily

FDA Grants Aro Biotherapeutics Rare Pediatric Disease Designation for Pompe Disease Therapy

September 16, 2022

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Aro Biotherapeutics for ABX1100, an experimental siRNA conjugate that targets the Gys1 gene in muscle for the treatment of Pompe disease.

Pompe disease is a rare and deadly disease that can manifest at any age of life, including infancy. It is characterized by debilitating muscle weakness that progresses over time and arises from a mutation in the acid alpha glucosidase enzyme that is responsible for the breakdown of glycogen in muscle cells.

ABX1100 is a novel approach to treating Pompe disease that aims to reduce glycogen buildup in the muscles. Aro Biotherapeutics anticipates entering clinical trials with ABX1100 in mid-2023.

“Pompe disease affects patients of all ages but can be particularly devastating in children. 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,” said Mittie Doyle, chief medical officer of Aro Biotherapeutics.

The FDA awards Rare Pediatric Disease designation to drugs treating rare pediatric diseases that are serious or life-threatening and affect patients from birth to 18 years of age. Sponsors that receive the designation from the FDA are entitled to receive a Pediatric Review Voucher (PRV) upon approval in a pediatric disease indication, giving the holder access to a priority review, reduced review time, and potential expedited product approval.

The vouchers are potentially lucrative because they are transferrable. They can be used to reduce the review time of any drug to six months from ten months. Most recently, Marinus Pharmaceuticals sold a PRV for $110 million in July to Novo Nordisk.

“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,” said Sue Dillon, co-founder and CEO of Aro Biotherapeutics.

Photo: Sue Dillon, co-founder and CEO of Aro Biotherapeutics

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.