苯丙酮尿症新型治疗药物pgvaliase-pqpz的药理与临床研究现状
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摘要
Pegvaliase-pqpz(商品名Palynziq)是由美国BioMarin制药公司研发的一种新型酶疗药物,是首个苯丙氨酸解氨酶法治疗成年患者苯丙酮尿症的药物。2018-05-24,美国食品和药品监督管理局(FDA)批准pegvaliase-pqpz用于成年苯丙酮尿症患者的治疗。本文对pegvaliase-pqpz的作用机制、药代动力学、药物相互作用、临床评价和安全性等进行综述。
Pegvaliase-pqpz( trade name Palynziq) is a new type of enzymatic drug developed by BioMarin Pharmaceuticals( USA),which is the first phenylalanine ammonia-lyase to treat phenylketonuria in adult patients. On May 24 2018,the US Food and Drug Administration( FDA)approved pegvaliase-pqpz for the treatment of adult patients with phenylketonuria. In this paper,the mechanism,pharmacokinetics,drug interaction,clinical evaluation,and safety of pegvaliase-pqpz were reviewed.
Pegvaliase-pqpz( trade name Palynziq) is a new type of enzymatic drug developed by BioMarin Pharmaceuticals( USA),which is the first phenylalanine ammonia-lyase to treat phenylketonuria in adult patients. On May 24 2018,the US Food and Drug Administration( FDA)approved pegvaliase-pqpz for the treatment of adult patients with phenylketonuria. In this paper,the mechanism,pharmacokinetics,drug interaction,clinical evaluation,and safety of pegvaliase-pqpz were reviewed.
引文
[1]FDA.Pegvaliase-pqpz[EB/OL].2018-05-24[2018-10-16].https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761079s000lbl.pdf.
[2]喻唯民,徐力,李晓雯,等.苯丙酮尿症研究十八年[J].中国医学科学院学报,2003,25(2):218-222.
[3]周忠蜀,李鹏.苯丙酮尿症的诊断和治疗进展[J].北京医学,2014,36(4):250-252.
[4]WALTER J H,WHITE F J,HALL S K,et al.How practical are recommendations for dietary control in phenylketonuria?[J].The Lancet,2002,360(9326):55-57.
[5]BROWN C S,LICHTER-KONECKI U.Phenylketonuria(PKU):A problem solved?[J].Mol Genet Metab Reports,2016,6(1):8-12.
[6]VIAU K S,WENGREEN H J,ERNST S L,et al.Correlation of age-specific phenylalanine levels with intellectual outcome in patients with phenylketonuria[J].J Inherited Metab Disease,2011,34(4):963-971.
[7]JURECKI E R,CEDERBAUM S,KOPESKY J,et al.Adherence to clinic recommendations among patients with phenylketonuria in the United States[J].Mol Genet Metab,2017,120(3):190-197.
[8]SARKISSIAN C N,SHAO Z,BLAIN F,et al.A different approach to treatment of phenylketonuria:Phenylalanine degradation with recombinant phenylalanine ammonia lyase[J].Proceed Nat Acad Sci,1999,96(5):2339.
[9]MAHAN K C,GANDHI M A,ANAND S.Pegvaliase:a novel treatment option for adults with phenylketonuria[J].Curr Med Res Opinion,2019,35(4):647-651.
[10]MARKHAM A.Pegvaliase:First hlobal approval[J].Bio Drugs,2018,32(4):391-395.
[11]THOMAS J,LEVY H,AMATO S,et al.Pegvaliase for the treatment of phenylketonuria:Results of a long-term phase 3 clinical trial program(PRISM)[J].Mol Genet Metab,2018,124(1):27-38.
[12]LONGO N,DIMMOCK D,LEVY H,et al.Evidence-and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria[J/OL].Genet Med,2018:e1-e27.2018-12-14[2019-03-21].https://www.nature.com/articles/s41436-018-0403-z.pdf.
[13]HARDING C O,AMATO R S,STUY M,et al.Pegvaliase for the treatment of phenylketonuria:A pivotal,double-blind randomized discontinuation Phase 3 clinical trial[J].Mol Genet Metab,2018,124(1):20-26.
[14]ZORI R,THOMAS J A,SHUR N,et al.Induction,titration,and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria[J].Mol Genet Metab,2018,125(3):217-227.
[15]LONGO N,ZORI R,WASSERSTEIN M P,et al.Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults:combined phase 2 outcomes through PAL-003 extension study[J].Orphanet J Rare Diseases,2018,13(1):108.
[16]THE A C O M,VOCKLEY J,ANDERSSON H C,et al.Phenylalanine hydroxylase deficiency:diagnosis and management guideline[J].Genet Med,2013,16(2):188.
[2]喻唯民,徐力,李晓雯,等.苯丙酮尿症研究十八年[J].中国医学科学院学报,2003,25(2):218-222.
[3]周忠蜀,李鹏.苯丙酮尿症的诊断和治疗进展[J].北京医学,2014,36(4):250-252.
[4]WALTER J H,WHITE F J,HALL S K,et al.How practical are recommendations for dietary control in phenylketonuria?[J].The Lancet,2002,360(9326):55-57.
[5]BROWN C S,LICHTER-KONECKI U.Phenylketonuria(PKU):A problem solved?[J].Mol Genet Metab Reports,2016,6(1):8-12.
[6]VIAU K S,WENGREEN H J,ERNST S L,et al.Correlation of age-specific phenylalanine levels with intellectual outcome in patients with phenylketonuria[J].J Inherited Metab Disease,2011,34(4):963-971.
[7]JURECKI E R,CEDERBAUM S,KOPESKY J,et al.Adherence to clinic recommendations among patients with phenylketonuria in the United States[J].Mol Genet Metab,2017,120(3):190-197.
[8]SARKISSIAN C N,SHAO Z,BLAIN F,et al.A different approach to treatment of phenylketonuria:Phenylalanine degradation with recombinant phenylalanine ammonia lyase[J].Proceed Nat Acad Sci,1999,96(5):2339.
[9]MAHAN K C,GANDHI M A,ANAND S.Pegvaliase:a novel treatment option for adults with phenylketonuria[J].Curr Med Res Opinion,2019,35(4):647-651.
[10]MARKHAM A.Pegvaliase:First hlobal approval[J].Bio Drugs,2018,32(4):391-395.
[11]THOMAS J,LEVY H,AMATO S,et al.Pegvaliase for the treatment of phenylketonuria:Results of a long-term phase 3 clinical trial program(PRISM)[J].Mol Genet Metab,2018,124(1):27-38.
[12]LONGO N,DIMMOCK D,LEVY H,et al.Evidence-and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria[J/OL].Genet Med,2018:e1-e27.2018-12-14[2019-03-21].https://www.nature.com/articles/s41436-018-0403-z.pdf.
[13]HARDING C O,AMATO R S,STUY M,et al.Pegvaliase for the treatment of phenylketonuria:A pivotal,double-blind randomized discontinuation Phase 3 clinical trial[J].Mol Genet Metab,2018,124(1):20-26.
[14]ZORI R,THOMAS J A,SHUR N,et al.Induction,titration,and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria[J].Mol Genet Metab,2018,125(3):217-227.
[15]LONGO N,ZORI R,WASSERSTEIN M P,et al.Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults:combined phase 2 outcomes through PAL-003 extension study[J].Orphanet J Rare Diseases,2018,13(1):108.
[16]THE A C O M,VOCKLEY J,ANDERSSON H C,et al.Phenylalanine hydroxylase deficiency:diagnosis and management guideline[J].Genet Med,2013,16(2):188.