Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome)

ABSTRACT

Introduction: Morquio A syndrome is a rare, autosomal recessive, lysosomal storage disorder caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase (GALNS). In 2014, the use of recombinant human GALNS, elosulfase alfa, was approved in the European Union, Canada, the United States, Australia, and Brazil for the treatment of Morquio A syndrome. Elosulfase alfa is administered intravenously once-weekly at a dose of 2.0 mg/kg.

Areas covered: This is a review of the efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, and other outcomes of elosulfase alfa treatment of patients with Morquio A. A discussion of other treatment considerations, limitations, and future directions in the use of elosulfase alfa is provided.

Expert commentary: Pharmacokinetic studies outside of clinical trials and in ‘real-world’ clinical settings need to be performed. We cannot currently predict which patient is going to respond well to enzyme replacement therapy; thus, all patients should be given the option to receive treatment for at least 12 months. Additionally, accurate biomarkers for evaluating disease state and drug responsiveness would greatly aid in the treatment of patients with Morquio A. In addition, improved and innovative daily lifestyle measures are greatly needed to adequately measure clinical response and true impact on quality of life.

KEYWORDS:

• Elosulfase alfa
• enzyme replacement therapy
• GALNS
• lysosomal storage disorder
• Morquio A
• MPS IVA
• mucopolysaccharidosis

Declaration of interest

CJ Hendriksz is Director of FYMCA Medical Ltd and consultant for Actelion, Amicus, Alexion, BioMarin, Inventiva, Sanofi, Genzyme and Shire. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Writing assistance was utilized in the production of this manuscript and performed by Latoya M Mitchell (Medical writing services) and Michelle R Rizzo (Editorial services) of PharmaWrite, LLC and funded by BioMarin Pharmaceutical Inc.

Additional information

Funding

Medical writing and editorial support was provided by PharmaWrite, LLC, and funded by BioMarin Pharmaceutical Inc.