Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
Cheryl Wong Po Foo, PhD, Director of Clinical Development, Astellas Gene Therapies, responsible for the advancement of the clinical development program for Pompe disease.
The Spark Therapeutics phase 1/2 gene therapy trial testing SPK-3006 to treat late onset Pompe disease has officially started with the first person being given their first (and theoretically only) dose.
Amicus Therapeutics has announced they have begun a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug combination, AT-GAA, to treat late onset Pompe disease (LOPD).
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
Patients enrolled in all clinical studies for AT-GAA for Pompe disease, including the PROPEL pivotal study, will continue to receive study drug.
Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA.