MYOZYME TEMPORARY ACCESS PROGRAM (MTAP)

Children'S Research Institute, Washington DC

Investigators

Linked publications & trials

Abstract

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Myozyme Temporary Access Program (MTAP) is to provide patients with Pompe disease in the US access to intravenous enzyme replacement therapy (ERT) with Myozyme produced from a scaled-up manufacturing process for a limited time until production at this scale is approved for commercial use by the FDA. Myozyme produced via this scaled-up process has been used in numerous clinical studies to date, including the ongoing clinical trial in patients with late-onset Pompe disease (AGLU02704) (CNMC is one site for this trial) and by all commercial patients outside the US. As is often the case with recombinant protein manufacture, the process was developed at a smaller volume, optimized, and subsequently scaled up to produce larger volumes. Genzyme is pursuing licensure of product produced from the scaled-up manufacturing process via a supplement to the existing Biologics License Application for Myozyme. This protocol, to allow adult patients who require larger doses of Myozyme to receive this large scale manufactured product, will also ensure an adequate supply of FDA-approved smaller volume produced Myozyme to be used for infantile and juvenile patients with Pomoe disease.