BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has received orphan drug status for an investigational enzyme replacement therapy to treat Pompe disease that was developed by ZyStor Therapeutics in Milwaukee, Wisconsin. Just two weeks ago, BioMarin bought ZyStor for $22 million in cash.
BioMarin is a publicly traded biopharmaceutical company in Novato, California, that sometimes partners with other drug developers on therapies for serious diseases and medical conditions. The California company promised to pay up to an additional $93 million for ZyStor if development, regulatory and commercial milestones for its leading therapy candidate are met.
That candidate is ZC-701 — now called BMN-701 by BioMarin — which is “a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA),” BioMarin said in a press release.
The therapy enables people with Pompe disease to use glycogen, a form of sugar stored for energy. Lacking the enzyme alpha glucosidase, glycogen accumulates in the cells of the body, disabling the heart and other muscles, and leading to death for infants and debilitation for adults, according to the National Institute of Neurological Disorders and Stroke (NINDS).
Pompe disease is rare — estimated at occurring only once in every 400,000 births, the NINDS said. The orphan drug designation from the Food and Drug Administration (FDA) gives BioMarin access to tax credits and marketing incentives to help pay for commercializing a therapy that has such a small market.
“Receiving orphan drug designation from the FDA for BMN-701 is a significant milestone for our Pompe program,” said Jean-Jacques Bienaime, BioMarin’s CEO, in his company’s statement.
Worldwide sales of replacement therapies for enzyme-related diseases were about $2.8 billion in 2008, according to ZyStor. Despite strong sales, existing drugs tend to have limited ability to target the lysosome — the compartments in cells that store the indigestible substance, in the case of Pompe disease, glycogen — so they require high infusion doses, the Milwaukee company said. The therapies also are difficult to make.
The current standard of care for Pompe disease is Genzyme Corp.’s Myozyme and Lumizyme, BioMarin said.
But based on pre-clinical evidence and manufacturing experience, BMN-701 could overcome the limitations of other enzyme replacements for the disease, ZyStor said. That’s because of ZyStor’s patented Glycosylation Independent Lysosomal Targeting (GILT) technology, which has the potential to deliver more enzyme to lysosomes than existing therapies.
“Relative to our internal candidate for Pompe … BMN-701 has a faster clinical development timeline, lower development costs, significantly lower cost of goods and lower capital investment,” Bienaime said two weeks ago when his company acquired ZyStor and its GILT technology.
ZyStor had submitted to the FDA an Investigational New Drug application for its Pompe disease therapy. The Wisconsin company also had made material to be used during a Phase I/II clinical study. BioMarin expects that study to begin in the first quarter of 2011.
At the time of its acquisition, Bienaime told securities analysts that ZyStor’s Milwaukee laboratory would remain open until the end of the year. ZyStor has seven employees, including Loren Peterson, the company’s president and chief executive, he said.
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