Enzyme Replacement Therapy (ERT)


Enzyme Replacement Therapies with Marketing Approval by the European Medicines Agency (EMA) 

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MPS I (Hurler Scheie & Scheie Disease)

Aldurazyme®, administered once weekly, has been approved in the European Union for the long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of MPS I, to treat the nonneurological manifestations of the disease. Aldurazyme was developed by BioMarin and Genzyme under a joint venture agreement that assigns commercial manufacturing responsibilities to BioMarin, and worldwide sales and marketing responsibilities to Genzyme. Aldurazyme is a weekly infusion lasting 3 – 4 hoursthat is usuallyadministered at home. Additional information can be obtained at www.aldurazyme.com

MPS II (Hunter Disease)

Elaprase®, is a a long term ERT for patients with a confirmed diagnosis of MPS II, where there is no cognitive decline. Elaprase has been approved for use in the European Union and was developed and is produced by Shire Human Genetic Therapies (formerly TKT). It is given as weekly infusions to replace the missing enzyme that Hunter disease patients fail to produce in sufficient quantities. Elaprase is a weekly infusion lasting 3 – 4 hours that is usually administered at home. Additional information can be obtained at www.shire.com 

MPS IVA (Morquio Disease)

Vimizim® is a long term ERT for patients with a confirmed diagnosis of MPS IVA, as approved for use in the European Union since April 2013. Vimizim was developed and is produced by BioMarin Pharmaceuticals Inc. and is given as a weekly infusion over a period of 3 – 4 hours. Additional information can be obtained at www.vimizim.com

MPS VI (Maroteaux Lamy Disease)

Naglazyme®, is the ERT for individuals with a confirmed diagnosis of MPS VI and has been approved for use in the European Union. It was developed and is produced by BioMarin Pharmaceuticals, Inc. Naglazyme is a weekly infusion lasting 3 – 4 hours that is administered at home. Additional information can be obtained at www.bmrn.com

Fabry Disease

Replagal® and Fabrazyme® are both Enzyme Replacement Therapies for the treatment of Fabry disease and both are approved in the European Union. Replagal was developed and is produced by Shire Human Genetic Therapies. Fabrazyme was developed and is produced by Genzyme. Replagal is an every other week infusion given over 40 minutes and administered at home. Fabrazyme is also an every other week infusion given over 2 – 4 hours and administered at home. For additional information please visit www.shire.com  and www.fabrazyme.com