Amid its latest run for FDA approval of its Gaucher disease drug tied into a collaboration with Pfizer ($PFE), Protalix BioTherapeutics ($PLX) has published details from its Phase III package that was denied approval early this year. The Israel-based biotech's drug, taliglucerase alfa, has been part of Pfizer's jump into the rare disease game in recent years, and the developer disclosed last month that U.S. regulators had accepted its resubmission for marketing approval.
Protalix has already released positive top-line data from its late-stage program on the treatment for the rare condition, for which Genzyme and Shire are already providing similar therapies. Yet today the company highlighted that the 31-patient pivotal trial met its primary endpoint, showing that patients in a 60 U/kg dosing group had mean reduction in spleen volume of 38% and those who got a 30 U/kg dose had a decrease in spleen volume of 26.9%. The study also saw positive results in the secondary endpoints of decrease in liver volume, increase in platelet count and improvements in hemoglobin levels.
"The sustained efficacy and safety that we observed among Gaucher patients receiving taliglucerase alfa in this study suggests that it is a valuable long-term treatment option for patients with Gaucher disease pending regulatory approval," Dr. Ari Zimran, director of the Gaucher Clinic in Shaare Zedek Medical Center, and the lead investigator of the study, said in a statement.
Now the company is expecting word from the FDA by Feb. 1 2012 on whether it's latest bid for approval will be granted. Regulators issued a complete response letter on the firm's earlier bid, asking for more data from trials for the treatment and additional details related to manufacturing.
- here's the Protalix release