Data to four years, in a phase II trial with the product in oral research of Genzyme Gauchers disease, suggests a significant improvement in all indicators.

Tartrate eliglustat, in the form of Tablet for oral administration, is under development in order to offer a more comfortable treatment alternative for adult patients with Gauchers Disease Type I, in addition to expanding treatment options to achieve individualized clinical objectives. The enzyme replacement therapy, which is administered via intravenous infusion, is currently the best treatment available for patients with Gauchers Disease Type I.

Genzyme has already announced that the phase II trial, of 52 weeks duration, with eliglustat tartrate had complied with its primary objective, seeking a clinically significant response in at least two of the three primary objectives (improvements in the size of the spleen, and hemoglobin and platelet levels) in patients in the study… Patients have continued to receive eliglustat tartrate, in the extension of the study, more than four years. Data from this group of patients suggest improvements continued or stabilised in all indicators:

-volumes of spleen and liver dropped from the start in an average of 63% and 28%, respectively.

-hemoglobin and platelet levels increased from the start in an average of 2.3 g/dL and 95%, respectively.

-all patients (100%) had been at least three of the four therapeutic goals haematological and visceral set for replacement therapy enzymatic.

-data also indicate a continued improvement of bone mineral density, as measured by bone densitometry, with an increase in the average 0.8 since the beginning T score, in the lumbar spine.

In the phase II study, adverse events reported and that they have been in more than two patients during the four years of treatment include: viral infections (six patients), infections in the urinary tract and respiratory tract suerperiores (four patients each) and nasofaringitis, sinusitis, arthralgia, pain in the extremities, headacheincreased blood pressure, study of abnormal nerve conduction, abdominal pain and diarrhea (three patients each). It reported ten adverse events related to drugs in eight patients. All related events were moderately.

In addition, Genzyme has completed the recruitment of three trials of phase III of this oral therapy in development. Together, these trials constitute the largest clinical program on Gauchers Disease ever developed, with patients from more than thirty countries. In total, more than 350 patients are participating in phase III studies.

The first trial of phase III, ENCORE, is a randomized study open for adult patients with Gauchers Disease of type I, designed to compare the tartrate eliglustat with Cerezyme ® (imiglucerasa). The study includes adult patients who have previously received enzyme replacement therapy for at least three years and have achieved their therapeutic objectives. The second essay, ENGAGE, is a study randomized, double blind, controlled by placebo patients with Gauchers Disease of type I without previous treatment for at least nine months before joining the study. Data from these studies pivotal registration should be available in the first half of 2013. A third trial, called EDGE, compares a daily dose of eliglustat with two daily doses tartrate.

About Gauchers Disease

Gaucher disease is a hereditary condition that affects fewer than 10,000 people worldwide. People with Gaucher disease lack the sufficient amount of an enzyme, β-glucosidase (erythropoietine), which breaks down a particular type of fat molecule. As a result, cells engrosadas by the accumulation of these (called Gaucher cells) lipids accumulate in various parts of the body, mainly in the spleen, liver and bone marrow. The accumulation of Gaucher cells can lead to the increase of the spleen and liver, anemia, excessive bleeding and bruising, bone disease and other signs and important symptoms. The most common form of disease of Gaucher (type I) does not usually affect the brain.

About eliglustat tartrate

Eliglustat, a similar innovative oral dosage glucosilceramida tartrate, is designed to partially inhibit the glucosilceramida synthase enzyme, resulting in a reduced production of glucosilceramida. The glucosilceramida is the substance that accumulates in the cells and tissues of patients with Gaucher’s disease. In preclinical studies, this molecule, developed by Dr. James a. Shayman, of the University of Michigan, has shown a high power and specificity. From its mechanism of action, which is independent of the genotype, eliglustat tartrate could be a potential therapy for all patients with type I Gaucher disease. The beginning of studies of phase II and III with tartrate eliglustat for Gauchers Disease was the continuation of a broad preclinical research effort and an ambitious program of phase I. Eliglustat tartrate was well tolerated during four years in the study of phase II and continues to present a safety profile that supports research in phase III studies. Eliglustat tartrate is a research product and any use of this product should be within the framework of clinical trials underway authorized. The promising preliminary results obtained so far are not determinants to assess the safety and efficacy of this product, which must be assessed and determined by the health authorities (agencies evaluators) based on the results of these studies.

About Genzyme, a company Sanofi

Genzyme has led the development and delivery of transformative therapies for patients with rare and debilitating diseases for more than thirty years. We reached our goals thanks to research of the highest quality and compassion and commitment of our employees. We focus on rare diseases and multiple sclerosis, and are committed to produce a positive impact on the lives of patients and families to whom we provide services. That is the objective that helps us guide and inspiration every day. The portfolio of transforming therapies of Genzyme, marketed in countries around the world, is the result of progress vital pioneers in medicine. As company Sanofi, Genzyme is benefiting from the scope and resources of one of the biggest pharmaceutical in the world, with a common commitment to improve the lives of patients.Genzyme ® and Cerezyme ® are registered trademarks of Genzyme Corporation. All rights reserved.