Novel Oral Compound Has Potential For Gaucher Disease

Armen Hareyan's picture

Genzyme reported that a preliminary analysis of data from an ongoing open-label Phase 2 clinical trial of its investigational oral therapy Genz-112638 showed that the compound produced a meaningful impact on key clinical manifestations of Gaucher disease. The results highlight the potential of this compound to become an innovative treatment option for Gaucher disease.

Genzyme's Cerezyme (imiglucerase for injection), the standard of care for patients with Type 1 Gaucher disease, is administered through intravenous infusions. The company is developing Genz-112638, a capsule taken orally, to provide a convenient treatment alternative for patients and to provide a broader range of treatment options for physicians.


The primary analysis period of the Phase 2 study of Genz-112638 is scheduled to conclude later this year, and the results for all trial participants will be available in the first quarter of 2009. The study included 26 adults with Type 1 Gaucher disease at medical centers in North America, South America, Europe and Israel. It was designed to evaluate the efficacy, safety and pharmacokinetics of the compound over one year. Nearly all study participants had completed six months of treatment when the preliminary analysis was conducted, and approximately half of the participants had completed one year of treatment.

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 percent among the 21 patients for whom data were available.