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新西兰扩大1型和3型戈谢病药物Cerezyme获取

来源:生物谷 2013-08-08 09:53

2013年8月7日讯 /生物谷BIOON/ --新西兰药品管理机构PHARMAC 8月7日宣布,已批准一项建议,从2013年9月1日起,扩大对赛诺菲(Sanofi)旗下健赞(Genzyme)戈谢病(Gaucher disease)药物Cerezyme(依米苷酶,imiglucerase)获取。

该建议的主题是,扩大1型和3型戈谢病患者对Cerezyme的获取。

Gaucher disease(戈谢病)是溶酶体糖脂贮积症中较常见的一种,为常染色体隐性遗传,是因溶酶体内的酸性β-葡萄糖苷酶(acidβ-glucosidase),又称葡萄糖脑苷脂酶 (glucocerebrosidase,GC)缺陷致病,使葡萄糖脑苷脂贮积在各器官的单核巨噬细胞系统中,形成Gaucher cell(戈谢细胞)。常表现为多系统的脂质沉积,累及骨髓、肝脾、骨骼及神经系统。(生物谷Bioon.com)

英文原文:New Zealand to widen access for type 1 and type 3 Gaucher disease drug

Article | 07 August 2013 Print This ShareThis

New Zealand’s Pharmaceutical Management Agency PHARMAC has announced the approval of a proposal to widen access to Gaucher disease drug Cerezyme (imiglucerase) from French drug major Sanofi’s (Euronext: SAN) Genzyme subsidiary, from September 1, 2013.

This proposal was the subject of a consultation letter dated June 26, 2013: a proposal to widen access to imiglucerase for type 1 and-type 3 Gaucher-disease

In summary, the effect of the decision is that:

? The access criteria will be widened to included funded access to imiglucerase for patients with type 3 Gaucher disease; and

? The maximum funded dose of imiglucerase will be increased from 15 iu/kg per month to 30 iu/kg per month for children with type 1 or type 3 Gaucher disease meeting certain criteria.

Details of the decision

These guidelines are intended to assist relevant practitioners in gauging which patients are likely to be approved for imiglucerase. In view of the complexity of Gaucher disease severity assessment, each application is thoroughly evaluated by the Gaucher Panel to determine the appropriate imiglucerase treatment. All requested studies should be carried out in line with the relevant professional guidelines. Patients with Gaucher disease who meet the following criteria may be eligible for initiation of imiglucerase treatment based on current clinical evidence.

Patients eligible for initial approval of Special Authority

1) The patient must have a diagnosis of symptomatic type 1 or type 3 Gaucher disease by the demonstration of:

? Specific deficiency of glucocerebrosidase in leukocytes or cultured skin fibroblasts; and

? Genotypic analysis

Histology and genotype tests to be supplied with the initial application once available. Baseline MRI whole body Short Tau Inversion Recovery (STIR) and serum chitotriosidase reports must be provided.

2) Patients who have Gaucher type 2 disease are not eligible for subsidized treatment. If a patient has a medical condition which significantly impacts on life expectancy or the treatment would not have a significant chance of causing an improvement in the patient’s condition, it is considered inappropriate to initiate therapy with imiglucerase.

3) Patients who receive government funded imiglucerase treatment must be willing to participate in the long term evaluation of the efficacy of the treatment, as approved, if necessary, by an ethics committee. Collated data collected may be made available to international investigators. Patient anonymity should be preserved.

4) Consent for data collection must be obtained from the patient and his/her legal guardian(s), where appropriate in line with any ethics committee process and/or procedural requirements.

5) Unless otherwise agreed by PHARMAC, imiglucerase shall not be subsidized at a dose exceeding 30 iu/kg/month rounded to the nearest whole vial.

6) The Gaucher Panel will consider applications and provide advice on the appropriate management of any other patients referred to it by PHARMAC.

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