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FDA Approves Genzyme’s Cerdelga™ (eliglustat) Capsules
Only First-Line Oral Therapy Approved for the Treatment of Adults with Gaucher Disease Type 1
Public Company Information:
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the U.S. Food and Drug Administration (FDA) has approved Cerdelga™ (eliglustat) capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. A small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for Cerdelga treatment. Cerdelga is expected to be available to patients within a month.
“Cerdelga is an important new option for people living with Gaucher disease type 1,” said Rhonda Buyers, CEO of the National Gaucher Foundation. “As enzyme replacement therapy is the standard of treatment for Gaucher disease, patients receive regular intravenous infusions for life. With FDA's approval of a first-line oral treatment, Cerdelga has the potential to be a valuable treatment option for people living with this serious disease.”
“The approval of Cerdelga is encouraging for Gaucher patients because it is a first-line oral treatment that has demonstrated a positive risk/benefit profile, making it important from both a scientific and a clinical perspective,” said Dr. Pramod Mistry, MD, PhD, Professor of Medicine and Pediatrics and the Director of National Gaucher Disease Treatment Center at Yale University School of Medicine. “While enzyme replacement therapies break down fatty deposits that build up in cells and cause a variety of symptoms, Cerdelga inhibits the accumulation of these fatty deposits in the first place.”
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.
“More than twenty years ago, Genzyme introduced the world’s first treatment for Gaucher disease. We are proud to build on this legacy and continue to improve Gaucher patients’ lives through ongoing research and new therapies,” said Genzyme President and CEO, David Meeker, M.D. “The approval of Cerdelga represents our unwavering commitment to the Gaucher patient community.”
The FDA approval was based on efficacy data from two positive Phase 3 studies for Cerdelga: one in patients new to therapy (Trial 1), and the other in patients switching from approved enzyme replacement therapies (Trial 2). The filing also incorporated four years of efficacy data from the Cerdelga Phase 2 study.
In Trial 1, improvements were seen across the following endpoints after 9 months on Cerdelga: spleen size, platelet levels, hemoglobin levels, and liver volume. Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over eighteen months.
Trial 2 met the pre-specified criteria for non-inferiority to an enzyme replacement therapy (imiglucerase), which was a composite endpoint of each of the following parameters: spleen volume, hemoglobin levels, platelet counts, and liver volume. Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over two years.
The most common adverse reactions (≥10%) are fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide synthase (IC50 = 10 ng/mL) with broad tissue distribution. It reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease. For more information, visit cerdelga.com.
See full prescribing information for more details about warnings and precautions and a complete list of adverse reactions.
Genzyme’s Case Managers provide individualized support services for Gaucher patients, including helping patients identify insurance options; referrals to financial assistance programs; assistance with treatment authorizations if necessary; assistance with appeals; and access to educational resources to help patients learn more about their disease. To reach a local Genzyme Case Manager, call 800-745-4447 (press option #3) Monday through Friday, from 8 a.m. to 6 p.m. ET, or visit our Patient & Caregiver Support Services online portal at www.genzymesupportservices.com.
Marketing applications for Cerdelga are under review by the European Medicines Agency (EMA) and other regulatory authorities.
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 a new 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
CERDELGATM (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.
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 the new Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
Forward Looking Statements
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