Pompe Disease Treatment Companies

Top Industry Leaders in the Pompe Disease Treatment Market

Pompe Disease Treatment Key Companies Disclaimer: List of key companies in no particular orderLatest Pompe disease Companies Update

September 2023: Amicus Therapeutics has just obtained formal clearance from the U.S. Food and Drug Administration (FDA) for the combination therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat). The medicine is intended for the treatment of adult individuals diagnosed with late-onset Pompe disease (LOPD) who do not exhibit improvement while undergoing their current enzyme replacement therapy (ERT). Its formulation is specifically developed to enhance the absorption into muscle cells. Once inside the cellular environment, Pombiliti can undergo appropriate processing to attain its fully active and mature state, facilitating the breakdown of glycogen. Opfolda functions as an enzyme stabilizer specifically engineered to mitigate enzyme instability in the bloodstream.

October 2023: Aro Biotherapeutics, a biotechnology firm at the clinical stage, made an announcement on the enrollment of the initial participant in their Phase 1 study for ABX1100. This trial focuses on Aro's primary Centyrin-siRNA product, which aims to cure Pompe Disease, a genetic disorder. ABX1100 is an innovative compound that introduces a novel strategy for mitigating the accumulation of glycogen, which can reach hazardous levels, within the muscles of individuals diagnosed with Pompe Disease. The Phase 1 trial, now underway in Canada, will begin with a segment including normal, healthy volunteers. This segment will consist of a placebo-controlled, double-blinded study, where participants will receive increasing doses of the treatment. The purpose of this study is to test the safety, tolerability, pharmacokinetics, and several pharmacodynamic biomarkers in order to demonstrate target engagement.

List of Pompe disease Key companies in the market