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Elosulfase alfa: First Global Approval

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Abstract

Elosulfase alfa (Vimizim™) is a recombinant form of N-acetylgalactosamine-6-sulfatase (GALNS) that was developed by BioMarin Pharmaceutical Inc. as an enzyme replacement therapy for patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Patients with MPS IVA have a GALNS deficiency, which results in serious musculoskeletal, cardiorespiratory and other system disturbances. Elosulfase alfa was approved by the US FDA on 14 February 2014 for the treatment of MPS IVA. The European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recently recommended that elosulfase alfa be approved for use in the EU in the same indication. Within the last year, the manufacturer has also filed applications for approval for the use of elosulfase alfa in MPS IVA in Brazil, Australia, Canada and Mexico. This article summarizes the milestones in the development of elosulfase alfa leading to its first global approval in MPS IVA.

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Disclosure

The preparation of this report was not supported by any external funding. During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. M Sanford and JH Lo are salaried employees of Adis, Springer SBM.

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Authors and Affiliations

  1. Adis R&D Insight, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, North Shore, 0754, Auckland, New Zealand

    Jin Han Lo

  2. Adis, Auckland, New Zealand

    Mark Sanford

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  1. Mark Sanford
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  2. Jin Han Lo
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Correspondence to Jin Han Lo.

Additional information

This profile has been extracted and modified from the Adis R&D, Insight drug pipeline database. Adis R&D Insight tracks drug, development worldwide through the entire development process, from discovery through pre-clinical and clinical studies to market launch.

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Sanford, M., Lo, J.H. Elosulfase alfa: First Global Approval. Drugs 74, 713–718 (2014). https://doi.org/10.1007/s40265-014-0210-z

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  • DOI: https://doi.org/10.1007/s40265-014-0210-z

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