Shire's rare-disease challenge is heating up. The company won European approval for a U.S. plant slated to produce Vpriv, a Gaucher disease treatment that aims to rival Genzyme's Cerezyme. The facility is still awaiting FDA approval, but it's another charge forward for Shire in its joust for market share.
The approval not only will create new capacity for Vpriv, but promises to open up space for increased production of Replagal, another drug that could steal scripts from Genzyme. Replagal treats Fabry disease, and even though it's not yet FDA-approved, it has gained ground in Europe with Genzyme fighting supply problems with its rival Fabrazyme. It's also been available to 140 U.S. patients under the FDA's emergency-use protocol. The agency has Replagal on a fast-track review now, with a decision expected this spring.
Genzyme recently won FDA and EMA approval for its new Framingham, MA, manufacturing plant, which will be turning out Fabrazyme at a faster rate. Sanofi ($SNY), which now owns Genzyme, says the Fabrazyme shortage won't be resolved overnight, but it does expect the supply wrinkles to smooth this year. U.S. patients, who've been on restricted doses, will begin to see some relief next month. And some analysts think the increased supplies of Fabrazyme will leach off some patients from Replagal.
- see the release from Shire
- get the Reuters story
ALSO: A group of Fabry disease patients sued the FDA, claiming that the agency's licensing process is unconstitutional, pointing out that Fabry patients in Europe experienced less rationing than U.S. patients did because Shire's Replagal was approved and available there as an alternative to Genzyme's scarce Fabrazyme. Report