ATB200-02

Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a combination therapy formerly known as AT-GAA, has been approved by the U.S. Food and Drug Administration (FDA) to treat certain adults with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT). Its developer, Amicus…

Treatment with the two-part investigational therapy AT-GAA improved walking ability and lung function for up to three years among adults with Pompe disease in a Phase 1/2 clinical trial. Barry Byrne, MD, PhD, from the University of Florida, presented the findings at the 2022 MDA Clinical & Scientific Conference,…

The European Medicines Agency has accepted applications from Amicus Therapeutics seeking approval of AT-GAA, a two-component investigational therapy for the treatment of Pompe disease. “The acceptance of these filings is an important step forward for people living with Pompe disease and their families in Europe. Patients need new…

The U.S. Food and Drug Administration (FDA) is reviewing applications from Amicus Therapeutics asking for the approval of AT-GAA as a treatment for late-onset Pompe disease (LOPD). Amicus initiated a rolling application for AT-GAA late last year, which allowed the company to submit portions of its application as…

Amicus Therapeutics is recruiting participants for the fourth group of its ongoing Phase 1/2 clinical trial evaluating the company’s investigative combination therapy AT-GAA. The trial (NCT02675465), called ATB200-02, will assess the safety, tolerability, and efficacy of AT-GAA in different groups of Pompe disease patients. The trial has treated…