首页 » FDA信息 » 15年磨一剑——赛诺菲口服戈谢病药物Cerdelga获FDA批准——将颠覆戈谢病市场格局


来源:生物谷 2014-08-21 10:16

2014年8月21日讯 /生物谷BIOON/ --赛诺菲(Sanofi)及旗下健赞(Genzyme)8月19日宣布,FDA已批准Cerdelga(eliglustat)胶囊,用作特定1型戈谢病(Gaucher disease)成人患者唯一的一线口服疗法。Cerdelga不适用于经基因检测证实对Cerdelga代谢更快或代谢速度不确定的少数患者。赛诺菲计划在未来一个月内将Cerdelga推向市场。






关于戈谢病(Gaucher disease):

戈谢病是一种常染色体隐性遗传所造成的葡糖脑苷脂沉积症,主要是因编码葡萄糖脑苷酯酶(gIucocerebrosidase) 的结构基因突变,导致该酶缺乏,致使巨噬细胞内的葡萄糖脑苷脂不能被进一步水解而堆积在溶酶体中,导致细胞失去原有的功能。这些病理性细胞在人体器官中的浸润会造成骨骼、骨髓、脾脏、肝脏和肺部的病变。


英文原文:FDA Approves Genzyme's Cerdelga™ (eliglustat) Capsules

Only First-Line Oral Therapy Approved for the Treatment of
Adults with Gaucher Disease Type 1

CAMBRIDGE, Mass.—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.

About Cerdelga

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.

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