In August, Sanofi's ($SNY) Genzyme won the FDA's blessing to take brand-new orphan drug Cerdelga into the U.S. Gaucher disease market, where the company hoped the pill could show up older, intravenous treatments. And now, it could be on its way to the same opportunity in the EU.
On Friday, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the oral treatment for use in patients with the rare disease, which affects fewer than 10,000 people worldwide.
Cerdelga will offer European patients--whose standard of care is the company's own injectable Cerezyme, according to Genzyme CEO David Meeker--"more choice in how to manage this serious disease," he said in a statement.
|Genzyme CEO David Meeker|
Cerezyme isn't the only competing med Cerdelga will have to take on in the European marketplace if the EMA follows up with a green light. Shire's ($SHPG) Vpriv and Actelion's ($ATLN) Zavesca are also on the market. But notably missing is Pfizer's ($PFE) Elelyso, which the EMA turned down at CHMP's suggestion in 2012 because of its similarity to Vpriv.
Elelyso is on the market in the U.S., however. But to combat the extra competition, Genzyme has set a competitive price tag for easier-to-administer Cerdelga. The drug costs about $300,000 a year--the average annual cost of Cerezyme infusions once every two weeks, it figures. "Our goal is to price this in a way that does not bias the medical decision-making process," Meeker said in August.
- read the release
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