CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Genzyme, a Sanofi company, today announced that it has received the 2015 Industry Innovation Award from the National Organization for Rare Disorders (NORD) in recognition of Cerdelga® (eliglustat) capsules. Genzyme was selected for the company’s work in bringing an additional treatment option to patients with Gaucher disease. Cerdelga is the only first-line oral therapy for certain adult Gaucher disease type 1 patients.
“Cerdelga is an important option for people living with Gaucher disease, and underscores Genzyme’s commitment to understand and respond to the needs of patients living with a rare disease,” said Peter L. Saltonstall, President and CEO, NORD. "We applaud Genzyme’s dedication to providing more choices to patients in rare disease communities and are pleased to present them with NORD’s 2015 Industry Innovation Award."
Genzyme has been researching an oral therapy for Gaucher disease for fifteen years, from early chemistry and preclinical research through clinical development. The Cerdelga clinical development program is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries.
"We are proud to receive this award. The approval of Cerdelga exemplifies our commitment to bring innovative therapies forward to improve patients’ lives through ongoing research,” said Genzyme’s Head of Rare Diseases, Richard Peters, MD, Ph.D.
This award marks the fourth time that Genzyme has been recognized by NORD for its contributions. The award will be presented tonight at NORD’s "Portraits of Courage Celebration" in Washington, DC.
The NORD Gala is an annual event at which researchers and others are honored for improving the lives of people with rare diseases. Each year NORD honors organizations that have made a positive contribution to further the needs of the patient community, and have inspired the public to do so as well.
NORD is a nonprofit organization established in 1983 to represent all individuals and families affected by rare diseases. It provides advocacy, education, support for research, and patient assistance programs to improve the lives of the 30 million Americans living with rare diseases.
About Gaucher Disease
Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, β-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms. The most common form of Gaucher disease, type 1, generally does not affect the brain.
About Genzyme’s Gaucher Disease Program
Driven by a long-term commitment to meeting unmet needs in the Gaucher community, Genzyme has been researching and developing treatments for those living with Gaucher disease for over 20 years. The company’s first FDA-approved therapy was the world’s first treatment for Gaucher disease. A few years later, Genzyme developed a next-generation enzyme replacement therapy for Gaucher disease, which is now the standard of care with unmatched years of efficacy and safety data. In 2014, Genzyme was the first to bring to market a first-line oral treatment for Gaucher disease, providing an additional treatment option for patients. Genzyme will continue to serve this community and remains committed to future advancements.
Cerdelga (eliglustat), a novel glucosylceramide analog given orally, was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease. The concept was initially developed by the late Norman Radin, PhD, from the University of Michigan. In pre-clinical studies, the molecule, developed with James A. Shayman, MD, also from the University of Michigan, showed specificity for glucosylceramide synthase. Following an extensive pre-clinical and early clinical research program, Cerdelga was studied in the largest Phase 3 clinical program ever conducted in Gaucher disease.
IMPORTANT SAFETY INFORMATION
Indications and Usage
CERDELGA® (eliglustat) capsules are indicated for the long-term treatment of adults with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. Patients who are CYP2D6 ultra-rapid metabolizers (URMs) may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect. A specific dose cannot be recommended for those patients whose CYP2D6 genotype cannot be determined (indeterminate metabolizers).
Important Safety Information
CERDELGA is contraindicated in the following patients due to the risk of significantly increased CERDELGA plasma concentrations which may result in prolongation of the PR, QTc, and/or QRS cardiac intervals that could result in cardiac arrhythmias: EMs or IMs taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor and IMs or PMs taking a strong CYP3A inhibitor.
Drugs that inhibit CYP2D6 and CYP3A may significantly increase the exposure to CERDELGA; Cerdelga dose adjustment may be needed, depending on metabolizer status. See section 7 of the full Prescribing Information for more details and other potentially significant drug interactions.
Because CERDELGA is predicted to cause increases in ECG intervals at substantially elevated plasma concentrations, use is not recommended in patients with pre-existing cardiac disease, long QT syndrome, or in combination with Class IA and Class III antiarrhythmic medications.
The most common adverse reactions (≥10%) for CERDELGA are: fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Only administer CERDELGA during pregnancy if the potential benefit justifies the potential risk; based on animal data, CERDELGA may cause fetal harm. Discontinue drug or nursing based on importance of drug to mother. CERDELGA is not recommended in patients with moderate to severe renal impairment or in patients with hepatic impairment.
To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation at (1-800-745-4447) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information, including patient Medication Guide, for additional important safety information.
Forward Looking Statements
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About Genzyme, a Sanofi Company
Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme’s portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more at www.genzyme.com.
Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
Genzyme® and Cerdelga® are registered trademarks of Genzyme Corporation. All rights reserved.
Genzyme Media Relations
Ingrid Mitchell, 617-768-6699