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Drugs

, Volume 77, Issue 11, pp 1247–1249 | Cite as

Cerliponase Alfa: First Global Approval

  • Anthony Markham
AdisInsight Report

Abstract

Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the progression of functional decline. This article summarizes the milestones in the development of cerliponase alfa leading to its first global approval in the USA for the treatment of motor function loss in paediatric patients ≥3 years of age with CLN2, and subsequent approval in the EU for CLN2 in all ages.

Notes

Compliance with Ethical Standards

Disclosure

The preparation of this review 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 author on the basis of scientific completeness and accuracy. A. Markham is a contracted employee of Adis, Springer SBM.

Additional information about this Adis Drug Review can be found at http://www.medengine.com/Redeem/8898F0604C065F70.

References

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    Williams RE, Adams HR, Blohm M, et al. Management strategies for CLN2 disease. Pediatr Neurol. 2017;69:102–12.CrossRefPubMedGoogle Scholar
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    BioMarin Pharmaceutical. BRINEURA (cerliponase alfa): US prescribing information. 2017. http://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761052lbl.pdf. Accessed 5 May 2017.
  3. 3.
    Eurpean Medicines Agency. Cerliponase alfa (Brineura): summary of product characteristics 2017. http://ec.europa.eu/health/documents/community-register/2017/20170530137963/anx_137963_en.pdf. Accessed 2 June 2017.
  4. 4.
    Gissen P, Schulz A, Specchio N, et al. Intracerebroventricular cerliponase alfa (BMN 190) in children with CLN2 disease: results from a phase 1/2, open-label, dose-escalation study (abstract). Dev Med Child Neurol. 2017;59(Suppl 1):7–8.Google Scholar
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    Schulz A, Specchio N, Gissen P, et al. Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study (abstract no. 302). Mol Genet Metab. 2017;120(1–2):S120.CrossRefGoogle Scholar

Copyright information

© Springer International Publishing Switzerland 2017

Authors and Affiliations

  1. 1.SpringerMairangi BayNew Zealand

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