Amicus Therapeutics, Inc. (NASDAQ:FOLD) posted topline results in treating LOPD (Late-onset Pompe disease) using its AT-GAA in phase 3, 52-week clinical trial. The US FDA previously granted breakthrough therapy designation for AT-GAA.
Seeks BLA submission for AT-GAA
The UK’s MHRA also awarded innovative medicine designation for AT-GAA. Amicus will plan to complete BLA (Biologics License Application) submission for its drug – AT-GAA after getting the nod from FDA in Q4 2021. It also plans for additional regulatory submissions in other geographies and the EU this year.
What is Pompe disease?
It is a glycogen storage disease Type II and caused due to insufficient functioning of enzyme – Lysosomal Acid α-1, 4-Glucosidase. The disease occurs due to mutation of the GAA Gene. In Pompe disease, the mutated GAA gene prevents the functioning of Acid α Glucosidase. It results in the buildup of glycogen within the cytoplasm and Lysosome and causes cellular damage and destruction. If it is not treated in time, it could cause heart failure.
The randomized, double-blind, 52-week phase 3 clinical trial is for assessing the tolerability, safety, and efficacy of the drug AT-GAA in treating Pompe disease compared to the standard care offered by alglucosidase alfa. 123 adult patients are enrolled for the Phase 3 clinical study and they could walk freely and breathe without any mechanical ventilation. The clinical trial is conducted on 5 continents and at 62 clinical sites spanning 24 nations.
In this clinical trial, two patients are administered with AT-GAA whereas one patient with alglucosidase alfa randomly. The clinical study of 117 patients engaged in the 52-week study is completed whereas 117 Pompe patients are voluntarily enrolled for long-term study and being treated solely with AT-GAA.
The data collected from pompe patients treated using AT-GAA shows positive and meaningful changes in the 52-week clinical trial. It has shown significant improvements in respiratory and musculoskeletal endpoints treated with AT-GAA compared to those treated with ERT. Therefore, Amicus’s AT-GAA has showed a higher potential to provide standard of care for people suffering from Pompe disease.
It improved respiratory and motor functions in patients suffering from Pompe disease. Therefore, Amicus’s AT-GAA provides an alternative treatment for people leading a life with Pompe disease.