Table 1. Approved enzyme replacement therapies available for the lysosomal storage disorders .
LSDs | Deficient enzyme | Inheritance | FDA approved ERT and Brand name |
MPS I (Hurler syn.) MPS II (Hunter syn.) MPS IV A (Morquio A syn.) MPS VI (Marateaux-Lamy syn.) |
α-L-iduronidase Iduronate sulfatase N-acetylgalactosamine 6-sulfatase N-acetylgalactosamine 4-sulfatase |
Autosomal X-linked Autosomal Autosomal |
Laronidase (Aldurazyme™)/ 2003-FDA, EMA Idursulfase (Elaprase™)/ 2006-FDA; 2007-EMA Elosulfase Alfa (Vimzim™)/ 2014-FDA Galsulfase (Naglazyme™)/ 2005-FDA; 2006-EMA |
Fabry disease | α-galactosidase | X-linked |
Agalsidase α (Fabrazyme™)/ 2001-EMA Agalsidase β (Replagal™)/ 2003-FDA, EMA |
Pompe diseas | α-glucosidase | Autosomal |
Aglucosidase (Myozyme™)/ 2006-FDA, EMA Aglucosidase (Lumizyme™)/ 2010-FDA |
Gaucher disease | β -glucocerebrosidase | Autosomal |
Aglucerase (Ceredase™)/ 1991-FDA Imiglucerase (Cerezyme™)/ 1994-FDA; 1997-EMA Velaglucerase (VPRIV™)/ 2010-FDA, EMA Taliglucerase (Elelyso™)/ 2012-FDA |
Lysosomal acid lipase deficiency | Lysosomal acid lipase | Autosomal | Sebelipase α (Kanuma™)/ 2015-FDA,EMA |