NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ — 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
disease. 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 (Myozyme/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 enzyme to lysosomes compared to
traditional mannose-6-phosphate targeted approaches using the
recently acquired GILT technology.”
About Pompe DiseasePompe disease, a lysosomal storage
disorder, is a progressive degenerative disease of the heart
muscle, diaphragm and skeletal muscle. It is caused by a
deficiency in the lysosomal enzyme acid alpha glucosidase which
leads to the accumulation of glycogen in myocyte lysosomes and
results in cell death. The incidence is one in 40,000 births.
There are two main forms of Pompe disease: adult onset with
an incidence of one in 57,000 births and infantile onset with an
incidence of one in
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