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FDA批准赛诺菲酶替代疗法Lumizyme用于全年龄段庞贝氏症(Pome)

2014-08-03     来源:生物谷
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2014年8月3日讯 /生物谷BIOON/ --赛诺菲(Sanofi)旗下健赞(Genzyme)7月31日宣布,FDA已批准扩大Lumizyme(α-葡萄糖苷酶,algucosidase alfa)适应症,用于所有年龄段或表型的庞贝氏症(Pompe)患者的治疗。此前,FDA于2010年5月仅批准Lumizyme用于8岁及以上晚发型庞贝氏症患者的治疗。

庞贝氏症(Pompe)是一种由溶酶体酸性α-葡萄糖苷酶(GAA)基因缺陷或功能障碍导致的渐进性、致衰性且往往致命性的罕见遗传性神经肌肉疾病,全球发病率估计为4万分之一,主要症状为心脏衰弱和骨骼肌无力,逐渐发展为呼吸衰竭,最终因呼吸衰竭死亡。该病因基因突变导致机体无法生成足够的酸性α-葡萄糖苷酶(GAA),而GAA为正常的肌肉功能所必需。心脏和肌肉细胞利用GAA将糖原转化为能量,当GAA缺乏,糖原会在骨骼肌、平滑肌和心肌等组织细胞中蓄积,最终使心脏和肌肉衰竭。

Lumizyme是一种水解性溶酶体糖原特异性酶,其作用机理是作为一种酶替代疗法,取代有缺陷的GAA,降低糖原在心脏和骨骼肌中的积累。

在美国,α-葡萄糖苷酶以2个不同的生产规模生产。160升规模(初试规模)生产的α-葡萄糖苷酶品牌名为Myozyme,4000升规模(最终生产规模)生产的α-葡萄糖苷酶品牌名为Lumizyme。根据所提供的部分生化和临床数据,FDA得出结论,认为以2种规模生产的α-葡萄糖苷酶(即Myozume和Lumizyme)具有可比性(comparable),α-葡萄糖苷酶的整体安全信息保持不变。

在美国以外,Lumizyme以商品名Myozyme上市销售,并已获超过65个国家批准。(生物谷Bioon.com)

英文原文:Genzyme Receives Label Expansion for Lumizyme? (alglucosidase alfa) in the United States for the treatment of Pompe Disease

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that the U.S. Food and Drug Administration (FDA) approved a supplement to expand the indication for Lumizyme? (alglucosidase alfa). Lumizyme manufactured at the 4000L scale is now indicated for all Pompe patients of any age or phenotype. The approval of this indication is now consistent with that of the rest of the world, where alglucosidase alfa manufactured at the 4000L is the only scale available. Previously, in the United States, Lumizyme had been approved only for patients with late onset Pompe disease.

“We are thankful to the entire Pompe community who has been on this journey to provide a sustainable, long-term option for Pompe patients in the United States,” said Genzyme President and CEO, David Meeker, M.D. “We are pleased we can now offer alglucosidase alfa produced at the 4000L scale to all patients in the US.”

In the United States, alglucosidase alfa is manufactured at two different production scales. Alglucosidase alfa manufactured at the 160L scale (initial pilot scale) has a brand name of Myozyme? (alglucosidase alfa)and alglucosidase alfa manufactured at the 4000L (final manufacturing scale) has a brand name of Lumizyme.

Based on the biochemical and clinical data provided as part of the submission, FDA concluded that alglucosidase alfa manufactured at both scales in the US (i.e., Lumizyme and Myozyme) are comparable. The overall safety profile of alglucosidase alfa remains unchanged.

Specific updates to the Lumizyme product label include:

Updated Indication: LUMIZYME? (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
Inclusion of safety and efficacy data from infantile-onset studies
Removal of the REMS program
Update to the boxed warning to include infantile-onset specific warning regarding fluid overload.
Updated to Pregnancy Category C classification
Lumizyme, marketed as Myozyme outside of U.S., is approved in more than 65 countries. For the complete Lumizyme label, visit?www.lumizyme.com.

About Pompe Disease

Pompe disease is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an estimated 1 in 40,000 people worldwide. This enzymatic defect results in the accumulation of glycogen primarily in muscle tissues that leads to muscle weakness, loss of respiratory function, and often premature death. Absent treatment, when symptoms occur in infancy, babies typically die within the first year of life. When symptoms occur in childhood or adulthood, patients often lose their ability to walk and require wheelchairs to assist with mobility and experience difficulty breathing as well as mechanical ventilation to breathe.

Important Safety Information for Lumizyme and Myozyme

INDICATION

LUMIZYME? (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).