DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/blfzp6/biomarins) has announced the addition of the "BioMarin's BMN-701 for Pompe Disease" report to their offering.
BioMarin is developing BMN-701 for the treatment of Pompe disease, a rare genetic lysosomal storage disorder caused by deficiencies in a lysosomal enzyme called acid alpha glucosidase (GAA). Pompe patients are currently treated with enzyme replacement therapy (ERT), whereby the deficient enzyme is replaced through periodic infusions of a recombinant human version (rhGAA). Genzyme's Myozyme and Lumizyme are the two ERTs currently on the market. However, muscle cells do not take up Myozyme and Lumizyme optimally, which may explain why some patients do not respond well to therapy. Thus, there is a need for improved products and improving uptake is one approach to overcome current limitations of available products.
BMN-701 utilizes a proprietary approach - glycosylation independent lysosomal targeting (GILT) - to enhance GAA uptake by muscle cells. It combines recombinant GAA with a fragment of the IGF-II peptide, which is the natural ligand to a muscle cell receptor, which mediates uptake of ERT. Pre-clinical data are compelling, showing significantly increased cellular uptake. The main question is whether increased uptake will also occur in patients, and whether it will lead to improved clinical outcomes.
BioMarin has generated phase I/II data and outlined its plan for a pivotal phase II/III study that should start later this year. In this report, we review the science, the rationale for BMN-701's development, the strength of the early clinical data, the regulatory pathway, and the commercial landscape for the product.
Key Topics Covered:
Pompe is a Lysosomal Storage Disorder
Clinical and regulatory Discussion
- 4s3 Bioscience
- AngioChem/Glaxo SmithKline
- PanGen Biotech
For more information visit http://www.researchandmarkets.com/research/blfzp6/biomarins