Amicus Therapeutics

Amicus Therapeutics announced positive preclinical data showing that its investigational gene therapy for Pompe disease is effectively delivered and leads to significant glycogen reduction at key tissues in mice, including the brain and spinal cord.

Pompe disease, an inherited lysosomal storage disorder, is much more common than once thought, say several experts speaking at the Muscular Dystrophy Association’s recent 2019 MDA Clinical and Scientific Conference in Orlando, Florida. For years, the incidence of Pompe was believed to be anywhere from 1 in…

Amicus Therapeutics received two U.S. patents for ATB200, a unique recombinant (lab-made) human acid alpha-glucosidase (rhGAA) enzyme, and the biologic component of the company’s investigational Pompe disease therapy AT-GAA. One patent covers the composition of matter for rhGAA; the second, the methods for making ATB200. The patents expire in 2035…

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Amicus Therapeutics’ lead therapy candidate for the treatment of late-onset Pompe disease,  AT-GAA (ATB200/AT221). The FDA’s decision makes AT-GAA the first investigational therapy for Pompe disease to receive this regulatory status. It also further…

On a Friday the 13th some 20 years ago, 15-month-old Megan Crowley was diagnosed with Pompe disease. A few weeks later, so was her newborn brother, Patrick. Even the doctor who diagnosed the two children didn’t know much about the disease. Before that March day in 1998, he had…

Amicus Therapeutics has dosed the first patient in the Phase 3 PROPEL clinical trial that aims to compare investigational therapy AT-GAA (ATB200/AT221) to standard treatment in patients with late-onset Pompe disease. The trial (NCT03729362) is still recruiting patients. Throughout 2019, approximately 100 participants who have been previously…

Amicus Therapeutics is planning to launch a pivotal trial to compare its investigational therapy AT-GAA (ATB200/AT221) to the current standard of care available for patients with Pompe disease. The new trial is expected to provide additional clinical data to support Accelerated Approval for AT-GAA by the U.S.

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…

April 15 was International Pompe Day, and Amicus Therapeutics helped to raise awareness about the disease, as well as Fabry disease, by participating in several activities throughout the month, which also is designated Fabry Disease Awareness Month. Fabry disease is an inherited lysosomal storage disorder caused by deficiency of…

Interim results a from Phase 1/2 clinical trial support the therapeutic benefits of investigational therapy combination ATB200/AT2221 in patients with Pompe disease. Participants in the study showed improvements in motor and respiratory function. The therapy also resulted in a durable decrease of muscle damage biomarkers. These results will be presented…