Enzyme Replacement/Gene Therapy

12 Drugs classified under this biological system

Enzyme Replacement Therapy. Laronidase 500 U / 5 ml.
VIAL: 1. See lit.
Long-term enzyme replace. ther. in
confirm. mucopolysaccharidos. I
(MPS I, alfa-I-iduronidase defic.) for nonneurolog. manifests. of the dis.

Enzyme Replacement Therapy. Imiglucerase 400 U/Vial.
VIAL (lyophilized pwdr. for infus.): 1.
See lit.
Long term enzyme replace. ther. in
confirmed diagn. type 1 Gaucher dis.

Alimentary tract and metabolism enzymes. Idursulfase 2 mg/ml.
VIAL (sol. for infus.): 1 x 3 ml. See lit.
Long-term tmt. Hunter Syndr. (MPS II).
C/I: Hypersens.

Enzymes. Taliglucerase Alfa 200 U/vial.
VIAL: 1× 200 U. The recomm. dosage for
tmt.-naïve adult &ped. pts. above 2 yrs.
of age and older is 60U/ kg of bdy. wt.
admin. every other wk. as a 60 to 120
min. IV. See lit.
Indic. for long-term enzyme replac. ther.
(ERT) for adult. & ped. pts. above 2 yrs.
with a confirm. diagnos. of Type 1
Gaucher dis.
C/I: Hypersens.

Enzyme Replacement Therapy. Agalsidase Beta 5 mg/ml, 35 mg/7ml.
PWDR. FOR INFUS: 1 x 5 mg/ml,
35 mg/ml. See lit.
Long-term enzyme replace. ther. in
confirmed diagnos. Fabry dis. (alfagalactosidase A defic.).

recombinant human lysosomal acid lipase. sebelipase 2 mg/ml.
VIAL Conc. for sol. for infus. 1 X 10 ml
Pts. with rapidly progress. LAL defic. presenting within the first 6 months of life: starting dose is either 1 mg/kg or 3 mg/kg admin. as IV infus. 1 X week, depending on the clin. status of the pt.
Ped. and adult pts. with LAL defic:1 mg/kg administered as IV infus. once every other week. See lit.
For long-term enzyme replacement ther.(ERT) in pts. of all ages with lysosomal acid lipase (LAL) deficiency.
C/I: Hypersens.