The approval of this indication is now consistent with that of the rest of the world, where alglucosidase alfa manufactured at the 4000L is the only scale available. Previously, in the United States, Lumizyme had been approved only for patients with late onset Pompe disease.
"We are thankful to the entire Pompe community who has been on this journey to provide a sustainable, long-term option for Pompe patients in the United States," said Genzyme President and CEO, David Meeker, M.D. "We are pleased we can now offer alglucosidase alfa produced at the 4000L scale to all patients in the US."
In the United States, alglucosidase alfa is manufactured at two different production scales. Alglucosidase alfa manufactured at the 160L scale (initial pilot scale) has a brand name of Myozyme (alglucosidase alfa)and alglucosidase alfa manufactured at the 4000L (final manufacturing scale) has a brand name of Lumizyme.
Based on the biochemical and clinical data provided as part of the submission, FDA concluded that alglucosidase alfa manufactured at both scales in the US (i.e., Lumizyme and Myozyme) are comparable. The overall safety profile of alglucosidase alfa remains unchanged.
Specific updates to the Lumizyme product label include:
– Updated Indication: LUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
– Inclusion of safety and efficacy data from infantile-onset studies
– Removal of the REMS program
– Update to the boxed warning to include infantile-onset specific warning regarding fluid overload.
– Updated to Pregnancy Category C classification
Lumizyme, marketed as Myozyme outside of U.S., is approved in more than 65 countries.