Excerpt from August 30, 2010 press release . . .
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.
“Receiving orphan drug designation from the FDA for BMN-701 is a significant milestone for our Pompe program. As part of their assessment for designation, the FDA determined that BMN-701 is sufficiently different from alglusidase alfa (MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More/Lumizyme) to allow for a unique orphan designation. For this reason, clinical superiority over alglusidase alfa will not be necessary to secure orphan exclusivity for BMN-701,” said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. “This 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 enzymeA protein that helps the body carry out chemical reactions. More to lysosomesA small part of the cell that acts like a recycling center, helping break down waste materials. More compared to traditional mannose-6-phosphate targeted approaches using the recently acquired GILT technology.”