BioMarin's Pompe disease treatment gets orphan-drug designation

NOVATO, Calif. BioMarin Pharmaceutical on Monday announced its investigational treatment for a lysosomal storage disorder has received orphan-drug designation from the Food and Drug Administration.

The investigational drug BMN-701, designed to treat Pompe disease, has been submitted to the FDA for regulatory approval. Pompe disease is a progressive degenerative disease of the heart muscle, diaphragm and skeletal muscle.

"This [designation] emphasizes our mission of developing innovative, products for orphan diseases with an unmet medical need. We believe BMN-701 has the potential to possibly deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches using the recently acquired GILT technology," said Jean-Jacques Bienaime, BioMarin CEO.