6月21日,BioMarin Pharmaceutical宣布,日本厚生劳动省(MHLW)批准Voxzogo(vosoritide)注射液上市,用于治疗所有年龄段儿童生长板尚未闭合的软骨发育不全。

BioMarin 高度赞扬康德弘翼的数统团队。作为BioMarin的合作伙伴,康德弘翼的数统团队长期为其提供数统业务专项服务外包(FSP),包括数据管理、数据库开发,与编程服务。特别值得一提的是,定制化数据库开发服务不仅满足客户对复杂方案设计的要求,同时也高度赋能客户提升整体数据库管理质量和效率,配合其顺利完成NDA的递交。

【BioMarin公告全文如下】

BioMarin
Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted
Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with
Achondroplasia, Whose Growth Plates are Not Closed.


No Lower Age Restriction for Treatment


Japan Accounts for Approximately Half of the 1,500 Patient Opportunity in APAC
Region 

SAN RAFAEL, Calif.June 21, 2022 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the registration of VOXZOGO® (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.  Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.

“We are delighted to offer
children in Japan of all ages with achondroplasia access to a treatment option
that addresses the underlying genetic mechanism of the condition,” said JJ.
“CNP was discovered as a natural regulator of bone growth in Japan in 1990, so
we are especially proud to be able to offer a therapeutic choice there. We look
forward to nurturing our partnerships with advocates and the achondroplasia
community in Japan and beyond.”

The MHLW in Japan based its
decision on the outcomes of a global Phase 3 randomized, double-blind,
placebo-controlled study evaluating the efficacy and safety of Voxzogo and the
long-term extension of this Phase 3 study as well as data from patients
participating in a Phase 2 randomized, double-Blind, placebo-controlled
clinical trial evaluating the safety and efficacy of Voxzogo in infants and
young children with achondroplasia, age 0 to <60 months.

In 2021, Voxzogo received approvals in the United
States, Europe and Brazil.

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