BioMarin Pharmaceuticals has announced that it has received orphan drug designation from the US Food and Drug Administration (FDA) for BMN-701 as a treatment of Pompe disease.
Pompe disease is a progressive degenerative disease of diaphragm, heart muscle and skeletal muscle that affects one in 40,000 people in the US.
BMN-701 is a combination of insulin-like growth factor 2 and alpha glucosidase.
An investigational new drug application for BMN-701 has been submitted and Phase I/II clinical trials are being planned in the first quarter of 2011.
BioMarin CEO Jean-Jacques Bienaime said that receiving the orphan drug designation was a significant milestone for the company’s Pompe programme.
Orphan drug designation is granted by the FDA to drugs that treat diseases affecting less than 200,000 people in the US.