Both Shire Pharmaceuticals and Protalix have rolled out a fresh barrage of promising late-stage data for their next-gen Gaucher disease drugs, with each taking another big step toward a market that delivers $1.2 billion a year to Genzyme. Analysts have been poring over the new data coming out of the Lysosomal Disease Network World Symposium in Miami, and they see some big trouble for Genzyme, which is reporting progress with its oral therapy for Gaucher disease.
"We maintain our thesis that Shire and Protalix will erode about one-third of Genzyme's Cerezyme market share over time," says Brian Abrahams, an analyst at Oppenheimer & Co, in a research note.
In a release, Protalix notes that the group which received a 60 U/kg dose of taliglucerase alfa demonstrated a "statistically significant mean reduction in spleen volume of 38.0% (p<0.0001) and the 30 U/kg group demonstrated a significant mean reduction in spleen volume of 26.9% (p<0.0001)."
Shire, which is facing a looming PDUFA deadline on February 28, also is touting its results in Phase III.
"The combined data presented today provides additional and compelling support for the long-term clinical efficacy and safety of velaglucerase alfa," said Pramod Mistry, a professor at Yale University School of Medicine. "The Gaucher community is very fortunate to have velaglucerase alfa as an option for patients."
Cerezyme and its two prospective competitors are all infused. Shire and Protalix--which is partnered with Pfizer--are likely to wage a war for market share by lowballing Cerezyme's $200,000 annual price tag. So even if Genzyme--which stumbled badly in 2009 as it dealt with a variety of woes--stays the market leader, it will likely be forced to offer a more competitive price.
Long-term, Genzyme has placed some big hopes in eliglustat tartrate, an oral drug that still faces a significant stretch of clinical work but has produced some striking results in a mid-stage trial.