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 고시번호(시행일자)
고시 제2017-193호(2017.11.1)
■ 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
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？ 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
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