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Development of gene therapies—lessons from nusinersen

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Abstract

The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the Food and Drug Administration (FDA), and use of FDA’s expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases.

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References

  1. Nusinersen (SPINRAZA) FDA reviews and label. 2016. Available at:http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/209531Orig1s000TOC.cfm.

  2. FDA Draft Guidance for Industry: Target Product Profile - A Strategic Development Process Tool. 2007. Available at:www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm080593.pdf.

  3. FDA Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics. 2014. Available at:http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf.

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Authors and Affiliations

  1. Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA

    L Xu, I Irony & W W Bryan

  2. Division of Neurology Products, Office of New Drugs, Center for Drug Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA

    B Dunn

Authors
  1. L Xu
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  2. I Irony
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  3. W W Bryan
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  4. B Dunn
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Corresponding author

Correspondence to W W Bryan.

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The authors declare no conflict of interest.

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Xu, L., Irony, I., Bryan, W. et al. Development of gene therapies—lessons from nusinersen. Gene Ther 24, 527–528 (2017). https://doi.org/10.1038/gt.2017.64

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  • DOI: https://doi.org/10.1038/gt.2017.64

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