Aro Biotherapeutics Receives FDA Rare Pediatric Drug Designation for ABX1100 for the Treatment of Pompe Disease

2022-09-15

合作优先审批siRNA

PHILADELPHIA--(BUSINESS WIRE)-- Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Drug (RPD) designation for ABX1100, an investigational Centyrin-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 breakdown of glycogen in muscle cells. ABX1100 is a novel approach to treating Pompe disease that aims to reduce glycogen buildup in the muscles. “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, MD, FACR, Chief Medical Officer of Aro Biotherapeutics. The FDA awards RPD 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 RPD 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. “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, Co-Founder and CEO of Aro Biotherapeutics. About Aro Biotherapeutics Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of diseases. For more information, visit .

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