Enzyme replacement therapy: Class Overview and Comparison
Quick answer: Enzyme replacement therapy are a class of medicines used for specific therapeutic indications. iMedic covers 7 enzyme replacement therapy substances. Below is a comparison table linking to detailed pages for each.
Enzyme replacement therapy on iMedic (7 substances)
| Substance | Primary indications | Mechanism | Common dose |
|---|---|---|---|
| Aldurazyme | Mucopolysaccharidosis I (Hurler/Scheie syndrome) | Laronidase, recombinant human α-L-iduronidase that degrades accumulated glycosam | 0.58 mg/kg IV infusion once weekly |
| Brineura | CLN2 disease (neuronal ceroid lipofuscinosis type 2) | Cerliponase alfa is a recombinant human tripeptidyl peptidase-1 enzyme delivered | 300 mg intraventricular infusion every 2 weeks |
| Cerezyme | Gaucher disease type 1, Gaucher disease type 3 | Imiglucerase is a recombinant analogue of human beta-glucocerebrosidase that hyd | 30-60 U/kg IV every 2 weeks |
| Kanuma | Lysosomal acid lipase deficiency, Wolman disease | Sebelipase alfa is a recombinant human lysosomal acid lipase that hydrolyzes cho | 1 mg/kg IV every other week (up to 3 mg/kg weekly in rapidly progressive disease) |
| Mepsevii | Mucopolysaccharidosis VII (Sly syndrome) | Vestronidase alfa is a recombinant beta-glucuronidase that replaces deficient en | 4 mg/kg IV every 2 weeks |
| Pombiliti | Late-onset Pompe disease | Recombinant human acid alpha-glucosidase (cipaglucosidase alfa) that replaces de | 20 mg/kg IV every 2 weeks (with miglustat) |
| Vimizim | Mucopolysaccharidosis type IVA (Morquio A syndrome) | Elosulfase alfa is a recombinant N-acetylgalactosamine-6-sulfatase that replaces | 2 mg/kg IV once weekly |
About Enzyme replacement therapy
Enzyme replacement therapy share a common mechanism of action and clinical use. Specific dosing, side effects, contraindications, and drug interactions vary between individual substances within the class. Click any substance above for full prescribing information and patient guidance.
Common considerations across the class
- Indication-specific selection: Different members may be preferred for different conditions or patient populations
- Dose equivalence: Members of the same class are not always interchangeable on a 1:1 dose basis
- Drug interactions: Class members often share interaction profiles (e.g., CYP enzyme effects) but individual variation matters
- Side effects: Some side effects are class-wide; others are substance-specific
- Contraindications: Individual contraindications may not generalize across the class
Always consult the prescribing information for the specific medicine prescribed and discuss with your clinician.
Frequently asked questions
What are Enzyme replacement therapy?
Enzyme replacement therapy are medicines that share a common mechanism of action used for specific therapeutic indications. iMedic currently covers 7 substances in this class with detailed pages for each.
Are all Enzyme replacement therapy interchangeable?
No. While medicines in the same class share a mechanism, they differ in potency, dosing, drug interactions, and tolerability. Switching between them is a clinical decision based on individual response, side effects, and treatment goals.
How do I choose between different Enzyme replacement therapy?
Selection depends on the specific clinical indication, patient factors (age, comorbidities, kidney/liver function, other medications), tolerability of side effects, cost, and clinician preference. This is a prescribing decision.
Are Enzyme replacement therapy available as generics?
Most well-established class members are available as generic alternatives, often substantially less expensive than brand-name versions while clinically equivalent. Newer members may still be brand-only.
Last reviewed: by iMedic Medical Editorial Team. Our editorial process.