Delving into the Latest Updates on BioMarin Pharmaceutical Japan K.K. with Synapse

概览

疾病领域	数量

内分泌与代谢疾病	2
神经系统疾病	1

排名前五的药物类型	数量

酶	1
重组多肽	1

排名前五的靶点	数量

NPRB(心房钠尿肽受体B型)	1
PAH(苯丙氨酸羟化酶)	1

关联

2

项与 BioMarin Pharmaceutical Japan K.K. 相关的药物

Vosoritide

伏索利肽

靶点

NPRB

作用机制

NPRB激动剂

在研机构

BioMarin Pharmaceutical, Inc. [+3]

原研机构

BioMarin International Ltd.

在研适应症

软骨发育不全 [+6]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

欧盟 [+3]

首次获批日期2021-08-26

Pegvaliase-PQPZ

靶点

PAH

作用机制

PAH激动剂

在研机构

BioMarin Pharmaceutical Japan K.K. [+2]

原研机构

BioMarin Pharmaceutical, Inc.

在研适应症

苯丙酮尿症

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2018-05-24

100 项与 BioMarin Pharmaceutical Japan K.K. 相关的临床结果

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0 项与 BioMarin Pharmaceutical Japan K.K. 相关的专利（医药）

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1

项与 BioMarin Pharmaceutical Japan K.K. 相关的文献（医药）

2023-11-01·Molecular Genetics and Metabolism

Two-year interim safety and efficacy of pegvaliase in Japanese adults with phenylketonuria

Article

作者: Shintaku, Haruo ; Kuwahara, Mitsuhiro ; Lee, Lawrence ; Ishige, Mika ; Ito, Tetsuya ; Hamazaki, Takashi

Phenylketonuria (PKU) is an inborn error of metabolism caused by deficiency of phenylalanine hydroxylase, resulting in high blood phenylalanine (Phe) concentrations with potential for impaired neurocognition. Pegvaliase, a pegylated recombinant phenylalanine ammonia lyase that metabolizes Phe, is approved for use in adults with PKU and high blood Phe despite prior management. In the Phase 3 PRISM studies conducted in the United States, pegvaliase induction/titration/maintenance dosing led to clinically meaningful and statistically significant blood Phe reductions versus placebo, with a manageable safety profile. Here we report the primary endpoint, change in blood Phe levels from baseline to Week 52, and 2-year interim efficacy and safety results (to Week 144; data cut-off March 31, 2022) of an ongoing, open-label study in a Japanese PKU population (JapicCTI-194,642). Participants were 12 adults with PKU from Japan aged 18-70 years with blood Phe levels >600 μmol/L. In Part 1, participants received subcutaneous 2.5 mg pegvaliase once weekly for 4 weeks (induction), followed by titration up to 20 mg/day, then dose adjustment to a maximum 40 mg/day to achieve blood Phe efficacy (≤360 μmol/L); this maintenance dose was continued to Week 52. In Part 2, participants continued pegvaliase with dose adjustments up to a maximum 60 mg/day for up to 168 weeks. Among 11 participants evaluable for efficacy, mean (standard deviation) blood Phe concentration decreased from 1025.9 (172.7) μmol/L at baseline to 448.3 (458.8) μmol/L at Week 52 (mean 57.5% decrease). Up to Week 104, all 11 (100%) efficacy-evaluable participants achieved blood Phe levels ≤600 μmol/L, 9 (81.8%) achieved ≤360 μmol/L, and 8 (72.7%) achieved ≤120 μmol/L. All 12 participants reported ≥1 adverse event (AE), most commonly injection site erythema and injection site swelling (n = 10, 83.3% each). The pegvaliase exposure-adjusted AE rate was 23.5 per person-years overall, 41.2 per person-years during induction/titration, and 13.5 per person-years during maintenance. All participants developed pegvaliase-induced antibody responses. There were no AEs leading to discontinuation, no deaths, and no anaphylaxis events. Although interim, these results support the use of pegvaliase in Japanese adults with PKU with elevated blood Phe levels and are consistent with results from the Phase 3 PRISM studies.

100 项与 BioMarin Pharmaceutical Japan K.K. 相关的药物交易

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100 项与 BioMarin Pharmaceutical Japan K.K. 相关的转化医学

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