Eliglustat tartrate경구제(품명: 세레델가캡슐84밀리그램)
기준 내용
허가사항 범위(CYP2D6 유전형 검사 상 CYP2D6 느린 대사자(PM, Poor metabolizer), 중간 대사자(IM, Intermediate metabolizer) 혹은 빠른 대사자(EM, Extensive metabolizer)로서 1형 고셔병(GD1, Gaucher disease 1) 성인 환자의 치료) 내에서 투여 시 요양급여를 인정함.
고시 신설 사유
신규 등재 예정인 1형 고셔병 치료제로 교과서, 가이드라인, 제외국 평가결과 등을 참조하여 치료 시작 전 CYP2D6 유전형 검사를 실시하여 허가범위에 해당되는 환자에게 인정토록 함.
관련문헌 등 12줄
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· Emery and Rimoin's Principles and Practice of Medical Genetics , Sixth Edition. 2013.
· Rhematology, Sixth Edition. 2015.
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· Clinical Genomics: Practical Applications in Adult Patient Care, First Edition. 2014.
· Belmatoug N, et al. Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe. Eur J Intern Med. 2016.
· Balwani M, et al. Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States. Molecular Genetics and Metabolism 117(2016)95-103.
· Pramod K. et al. Effect of Oral eliglustat on Splenomegaly in patients with Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial(허가임상). JAMA 2015 Feb 17; 313(7):695-706
· Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase3, randomised, open-label, non-inferiority trial(ENCORE). Lancet 2015 Jun 13; 385(9985):2355-62
· Cox TM, et al. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017 Feb 6
· Ibrahim J, et al. Clinical response to eliglustat in treatment-naive patients with Gaucher disease type 1: Post hoc comparison to imigluceerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. Molecular Genetics and Metabolism Reports. 2016 Jun 27;8:17-9.
· NICE(2017.5.)
출처: 건강보험심사평가원 보험인정기준 고시 고시 제2017-193호(약제) · 심평원 원문 보기. 참고용 정보이며 청구·심사의 최종 판단은 고시 원문과 심평원 기준을 따릅니다.