Which Enzyme Replacement Therapy are available?

This directory covers 6: Myozyme, Elaprase, Naglazyme, Vimizim, Lamzede, Kanuma.

Enzyme Replacement Therapies

Also called ERT, lysosomal storage disorder treatments

Some rare inherited diseases are caused by a single missing enzyme, so the substance it should break down builds up in cells until organs fail. Enzyme replacement therapy supplies a manufactured version of the missing enzyme by regular infusion — usually every one or two weeks, for life. It does not cure the underlying genetic fault, but it can halt or slow the damage.

What this class is used for

Pompe disease, Hunter syndrome, Maroteaux-Lamy syndrome, Morquio A, alpha-mannosidosis, LAL deficiency

6 Enzyme Replacement Therapy in this directory

Myozyme

Alglucosidase Alfa

An intravenous enzyme replacement therapy used to treat patients with Pompe disease.

Elaprase

Idursulfase

An intravenous enzyme replacement therapy used to treat Hunter syndrome.

Naglazyme

Galsulfase

An intravenous enzyme replacement therapy used to treat Mucopolysaccharidosis VI.

Vimizim

Elosulfase Alfa

An intravenous enzyme replacement therapy used to treat Morquio A syndrome.

Lamzede

Velmanase Alfa

An intravenous enzyme replacement therapy used to treat alpha-mannosidosis.

Kanuma

Sebelipase Alfa

An intravenous enzyme replacement therapy used to treat lysosomal acid lipase deficiency.

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Class descriptions are written by the Priya Life Science editorial team. Individual drug pages combine that summary with live label, approval, manufacturer and shortage data from the U.S. FDA via the openFDA API. This page is general information and is not medical advice — it is not exhaustive, drugs within a class are not automatically interchangeable, and approvals and brand names differ between the US, EU/Ireland (EMA/HPRA) and other regions. Always consult the official prescribing information and your clinician or pharmacist. Related: Drug Shortages Tracker · FDA Approvals · All drug comparisons