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The Pharmaceutical Development of rhDNase (Dornase Alpha) for the Treatment of Cystic Fibrosis

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Mucosal Delivery of Biopharmaceuticals

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Abstract

This review summarizes the development of rhDNase as a pharmaceutical for pulmonary delivery including development and stability assessment of a formulation, compatibility with jet nebulization, characterization of the aerosols produced by jet nebulization, and compatibility and storage stability of the final drug product configuration in blow-fill seal plastic ampoules. These evaluations supported the eventual health authority registration under the brand name Pulmozyme®. More recent life-cycle development efforts have involved performing in vitro technical feasibility studies to determine whether the existing drug product formulation of Pulmozyme® can be delivered by a portable electronic mesh nebulizer without any impact on the molecular, chemical, and physical attributes of product quality and with similar dosage as those originally used to register the drug. These studies illustrate the complexity of combination product development and life-cycle management to maintain the modernity of product delivery systems.

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Authors and Affiliations

  1. Genentech, Late Stage Pharmaceutical Development, San Francisco, CA, USA

    Steven J. Shire & Thomas M. Scherer

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  1. Steven J. Shire
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  2. Thomas M. Scherer
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Correspondence to Thomas M. Scherer .

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Editors and Affiliations

  1. IINFACTS - Instituto de Investigação e Formação Avançada em Ciências e Tecnologias da Saúde, Instituto Superior de Ciências da Saúde-Norte, CESPU, Gandra, Portugal

    José das Neves

  2. NEWTherapies Group, INEB – Instituto de Engenharia Biomédica, Porto, Portugal

    Bruno Sarmento

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Shire, S., Scherer, T. (2014). The Pharmaceutical Development of rhDNase (Dornase Alpha) for the Treatment of Cystic Fibrosis. In: das Neves, J., Sarmento, B. (eds) Mucosal Delivery of Biopharmaceuticals. Springer, Boston, MA. https://doi.org/10.1007/978-1-4614-9524-6_20

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