The FDA have given a thumbs down to Genzyme's request for permission to sell its Pompe disease drug Myozyme manufactured in an Allston, MA plant. Genzyme is already manufacturing the drug in a smaller Framingham-based plant and wants to ramp up production at the larger Allston location. But the FDA ruled that the Allston-produced Myozyme must "be classified as [a different product] because of differences in the carbohydrate structures of the molecules." The FDA said Genzyme has to submit a separate BLA to gain approval for Myozyme produced on a larger scale.
This rejection underscores the difficulty biosimilars face in the United States. "The FDA decision...suggests that regulators may be reluctant to approve any generic versions of biologic drugs...without clinical data proving the drugs are at least as safe and effective as the originals if there are even slight differences in the compounds," observes the Boston Globe. Currently, the U.S. has no pathway to quickly approve biosimilar drugs.
As Genzyme points out, Myozyme production has already been scaled up in 40 other countries. "Based on the global clinical experience of nearly 900 patients of all ages currently receiving Myozyme produced at the larger scale...Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe," the company said in a statement. The company expects the FDA to act on the application by the end of the year.
- see this releaseÂ from Genzyme
- read the Boston Globe article
ALSO: Genzyme is opening a major new R&D center in Beijing. The facility will house 350 employees and will cost an estimated $90 million. Researchers in the Beijing plant will develop drugs in four focused areas: orthopedics, transplant and immune disease, oncology, endocrinology and cardiovascular disease. According the the release, the R&D facility "is part of Genzyme's ongoing global expansion and commitment to establishing a long-term presence in China." Report
Manufacturing woes plague launch of Myozyme
Genzyme wins Myozyme OK
Myozyme approval leads to rich royalty pact