A drug candidate for Pompe disease (PD) recently received the Rare Pediatric Drug (RPD) designation from the US Food and Drug Administration (FDA), according to a recent press release.

ABX1100 is a centyrin-small interfering RNAs (siRNAs) conjugate under experimental research as a novel treatment option for patients with PD. Gaining the RPD is an important step while developing any new drug since it may accelerate the process of reviewing and potentially approving this therapy by making it eligible to receive a Pediatric Review Voucher. RPDs remain reserved for life-threatening or severe entities affecting newborns and individuals up to 18 years of age.

The designation was announced in an Aro Biotherapeutics press release.

Continue Reading

Read more about PD therapies

These advances represent promising news for patients with PD. PD is an uncommon inherited disease. It may present at any age, including in the newborn and infancy stages. The most prominent clinical features include muscle weakness of debilitating nature that progress over time.

The siRNA molecules target specific genes to treat genetic diseases. Centyrin-siRNA specifically targets the GYS1 gene, which is mutant in patients with PD.

Mutations in the GYS1 gene result in a deficient acid alpha-glucosidase enzyme that is unable to properly break down glycogen in myocytes, generating a continuous buildup that impairs the contractile function.


Aro Biotherapeutics receives FDA rare pediatric drug designation for ABX1100 for the treatment of Pompe disease. News release. Aro Biotherapeutics; September 15, 2022.