Nanoparticle-based drug delivery systems: a commercial and regulatory outlook as the field matures

ABSTRACT

Introduction: Nanomedicine has emerged as a major field of academic research with direct impact on human health. While a first generation of products has been successfully commercialized and has significantly contributed to enhance patient’s life, recent advances in material design and the emergence of new therapeutics are contributing to the development of more sophisticated systems. As the field matures, it is important to comprehend the challenges related to nanoparticle commercial development for a more efficient and predictable path to the clinic.

Areas covered: The review provides an overview of nanoparticle-based delivery systems currently on the market and in clinical trials, and discuss the principal challenges for their commercial development, both from a manufacturing and regulatory perspective, to help gain understanding of the translational path for these systems.

Expert opinion: Clinical translation of nanoparticle-based delivery systems remains challenging on account of their 3D nanostructure and requires robust nano-manufacturing process along with adequate analytical tools and methodologies. By identifying early enough in the development the product critical attributes and understanding their impact on the therapeutic performance, the developers of nanopharmaceuticals will be better equipped to develop efficient product pipelines. Second-generation products are expected to broaden nanopharmaceutical global market in the upcoming years.

KEYWORDS:

• Clinical translation
• drug development
• FDA approval
• nano-manufacturing
• nanomedicine
• marketed nanoparticle
• nanopharmaceutical
• nanosimilar
• regulatory

Article highlights

• Nanoparticle-based delivery systems have emerged as a major field of research and commercialized first-generation products have significantly improved human health.

• The market of nanopharmaceuticals is expected to grow on account of the arrival of second-generation products and nanosimilars.

• As the field matures, it is important to realize the challenges related to nanoparticle commercial development for a more efficient and predictable path to the clinic.

• The 3D nano-structure of nanopharmaceuticals makes consistent manufacturing and quality control difficult.

• Understanding the critical physico-chemical attributes that are responsible for the therapeutic performance is essential for successful clinical translation and nanosimilar development.

This box summarizes key points contained in the article.

Acknowledgments

The authors thank Dr M. W. Tibbitt for feedback on the manuscript.

Declaration of interest

H Ragelle is funded by the Helmsley Foundation. DG Anderson is a consultant with Shire Phamaceutics and CRISPR Tx. F Danhier is a postdoctoral researcher from the Fonds National de la Recherche Scientifique FNRS. R Langer holds equity in Alnylam, Moderna, Kala and Selecta. The authors have 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.

Additional information

Funding

This work was supported by the Leona M. and Harry B. Helmsley Charitable Trust and Fonds De La Recherche Scientifique – FNRS.