The preliminary analysis showed that Genz-112638 produced promising efficacy results at six months and that these results continued to improve through one year of treatment. At six months, spleen volumes had decreased from baseline by a mean of 27% among the 21 patients for whom data were available. Spleen volumes had decreased by 40% among 11 patients with available data at one year.
At six months, hemoglobin levels had increased from baseline by a mean of 0.9 grams per deciliter of blood among 17 patients for whom data were available. Hemoglobin levels had increased by 1.3 grams per deciliter among 13 patients with available data at one year. Platelet counts increased from baseline by a mean of 18% among 17 patients treated for six months and by 34% among 13 patients with available data at one year.
Chitotriosidase levels decreased from baseline by a mean of 30% at six months among 20 patients and by 50% among 12 patients treated for one year. Chitotriosidase commonly serves as a biomarker of Gaucher disease burden, allowing physicians to monitor patient response to treatment.
David Meeker, Genzyme’s president of lysosomal storage disorder therapeutics, said: “The data we collect from this study and from the Phase III program will provide guidance on the roles that this compound may play in treating and maintaining patients with Gaucher disease.”