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首页 » FDA信息 » FDA批准辉瑞Elelyso治疗1型戈谢病儿科患者

FDA批准辉瑞Elelyso治疗1型戈谢病儿科患者

来源:生物谷 2014-08-29 09:55

2014年8月28日讯 /生物谷BIOON/ --辉瑞(Pfizer)和Protalix生物治疗公司8月28日宣布,FDA已批准Elelyso(taliglucerase alfa)注射液用于1型戈谢病儿科患者的长期治疗。此次获批,使Elelyso成为可同时用于1型成人和儿科患者的长期酶替代疗法(ERT)。

Elelyso的儿科疗效和安全性在2项临床试验中进行了评估,涉及14例1型戈谢病儿科患者。其中一项安慰剂对照研究涉及9例患者,研究中Elelyso成功稳定了病情;另一项研究涉及5例儿科患者,从酶替代疗法Cerezyme(注射用依米苷酶)转向Elelyso治疗,病情成功稳定。

此前,FDA已于2012年5月批准Elelyso用于1型戈谢病成人患者。该药是一种酶替代疗法,每2周注射一次。Elelyso也是FDA批准的首个基于植物细胞表达系统的药物,采用基因工程改造的胡萝卜细胞生产。

本月20日,赛诺菲(Sanofi)口服戈谢病药物Cerdelga(eliglustat)获FDA批准,该药的研发花费了足足15年时间。目前,酶替代疗法(ERT)是戈谢病的标准治疗方案。酶替代疗法(ERT)能够降解沉积在细胞中的脂肪沉积物,而Cerdelga则能够直接抑制脂肪沉积物在细胞中的积累。分析师预计,Cerdelga作为首个口服治疗药物,将完全颠覆当前依赖注射型药物的戈谢病市场格局,将于辉瑞自己的Cerezyme、夏尔(Shire)的Vpriv和辉瑞的Elelyso展开竞争。

详见《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》。

戈谢病(Gaucher disease)是一种罕见遗传性疾病,是由常染色体隐性遗传所造成的葡糖脑苷脂沉积症,患者体内无法产生足够的葡萄糖脑苷酯酶(GCD),该酶的不足导致脂肪物质沉积在脾、肝、肾等器官内,主要体征为肝脏或脾脏损害、贫血、血小板低和骨骼问题。目前,全球仅有1万名戈谢病患者,美国患者总数约为6000人。

英文原文:Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease

Thursday, August 28, 2014 - 1:15pm EDT
 
Pfizer Inc. (NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX, TASE:PLX) announced today that the U.S. Food and Drug Administration (FDA) approved ELELYSO™ (taliglucerase alfa) for injection for pediatric patients. ELELYSO is therefore now indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.
 
"The approval of ELELYSO to treat pediatric patients with Type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease,” said Rory O’Connor, Senior Vice President, Global Medical Affairs, Global Innovative Pharma Business, Pfizer Inc. “This pediatric indication, along with the recent announcement that ELELYSO received kosher certification by the Orthodox Union (OU), reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”
 
The safety and efficacy of ELELYSO were assessed in fourteen pediatric patients with Type 1 Gaucher disease in two clinical trials. The first trial consisted of nine patients in a 12-month, multi-center, double-blind, randomized study in treatment-naïve patients aged two to 13 years. At the end of the 12-month study, therapeutic efficacy of ELELYSO was demonstrated, as measured by a decrease in spleen and liver volume and an increase in platelet count. A second trial consisted of 5 pediatric patients aged 6 to 16 years who were switched from imiglucerase to ELELYSO. The trial was a 9-month, multi-center, open-label, single-arm study in patients who had been receiving treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60 units/kg every other week for a minimum of 2 years. ELELYSO was administered for 9 months at the same dose as each patient’s previous imiglucerase dose. If needed, adjustment of dosage was allowed during the study in order to maintain stability of clinical parameters. Mean spleen and liver volume, platelet count and hemoglobin value remained stable through 9 months of ELELYSO treatment.
 
The recommended dosage of ELELSYO for treatment-naïve adult and pediatric patients four years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion.
 
Patients previously treated on a stable dosage of imiglucerase are recommended to begin treatment with ELELYSO at that same dosage when they switch from imiglucerase to ELELYSO. Dosage adjustments can be made based on achievement and maintenance of each patient’s therapeutic goals.
 
Serious hypersensitivity reactions, including anaphylaxis, have occurred in some patients treated with ELELYSO. The most common adverse reactions for ELELYSO in clinical trials were itching, flushing, headache, joint pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea and rash. Vomiting occurred more often in pediatric patients than adults.
 
“While Type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood,” said Paige Kaplan, MB, BCh, Section of Biochemical Genetics (Metabolic Diseases), Children's Hospital of Philadelphia. “It is important that children with this disease have access to a range of FDA-approved treatment options that are effective.”
 
As part of its ongoing commitment to helping address the unmet medical needs of people with rare diseases, Pfizer also offers a specialized support program -- called Gaucher Personal Support (GPS) -- for people living with Gaucher disease. Pfizer GPS is a one-stop resource for personalized patient support and specialty pharmacy services for patients and healthcare professionals. Pfizer GPS employs a dedicated and caring team of healthcare specialists who are available to help patients with Gaucher disease and their caregivers with reimbursement assistance, coordinating and locating infusion services and ongoing pharmacy support. Using Pfizer GPS, patients and healthcare professionals can access all support services 24/7 using one toll-free phone number, 1-855-ELELYSO (1-855-353-5976).
 
ELELYSO for injection is supplied as 200 units per vial and is available by prescription only.
 
INDICATION
ELELYSO™ is indicated for long-term enzyme replacement therapy (ERT) for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.

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