Development of gene therapies—lessons from nusinersen

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. Access through your institution Buy or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution Subscribe to this journal Receive 6 print issues and online access $259.00 per year only $43.17 per issue Learn more Buy this article * Purchase on SpringerLink * Instant access to full article PDF Buy now Prices may be subject to local taxes which are calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support SIMILAR CONTENT BEING VIEWED BY OTHERS ANALYSIS AND COMPARATIVE EVALUATION OF EXPEDITED PROGRAMS FOR GENE THERAPY PRODUCTS: INSIGHTS FROM THE UNITED STATES, THE EUROPEAN UNION, JAPAN, AND SOUTH KOREA Article 10 January 2024 APPROVAL AND THERAPEUTIC VALUE OF GENE THERAPIES IN THE US AND EUROPE Article 08 November 2023 ETHICAL CHALLENGES FOR A NEW GENERATION OF EARLY-PHASE PEDIATRIC GENE THERAPY TRIALS Article 07 July 2021 REFERENCES * Nusinersen (SPINRAZA) FDA reviews and label. 2016. Available at:http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/209531Orig1s000TOC.cfm. * FDA Draft Guidance for Industry: Target Product Profile - A Strategic Development Process Tool. 2007.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. * 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. Download references AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * 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 * 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 * L Xu View author publications You can also search for this author inPubMed Google Scholar * I Irony View author publications You can also search for this author inPubMed Google Scholar * W W Bryan View author publications You can also search for this author inPubMed Google Scholar * B Dunn View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence to W W Bryan. ETHICS DECLARATIONS COMPETING INTERESTS The authors declare no conflict of interest. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE 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 Download citation * Received: 13 March 2017 * Revised: 13 July 2017 * Accepted: 18 July 2017 * Published: 24 July 2017 * Issue Date: September 2017 * DOI: https://doi.org/10.1038/gt.2017.64 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content: Get shareable link Sorry, a shareable link is not currently available for this article. Copy to clipboard Provided by the Springer Nature SharedIt content-sharing initiative

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. Access through your institution Buy


or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution Subscribe to this journal Receive 6 print issues and online


access $259.00 per year only $43.17 per issue Learn more Buy this article * Purchase on SpringerLink * Instant access to full article PDF Buy now Prices may be subject to local taxes which


are calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support SIMILAR CONTENT BEING VIEWED BY OTHERS


ANALYSIS AND COMPARATIVE EVALUATION OF EXPEDITED PROGRAMS FOR GENE THERAPY PRODUCTS: INSIGHTS FROM THE UNITED STATES, THE EUROPEAN UNION, JAPAN, AND SOUTH KOREA Article 10 January 2024


APPROVAL AND THERAPEUTIC VALUE OF GENE THERAPIES IN THE US AND EUROPE Article 08 November 2023 ETHICAL CHALLENGES FOR A NEW GENERATION OF EARLY-PHASE PEDIATRIC GENE THERAPY TRIALS Article 07


July 2021 REFERENCES * Nusinersen (SPINRAZA) FDA reviews and label. 2016. Available at:http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/209531Orig1s000TOC.cfm. * 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. *


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. Download references AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * 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 * 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 * L Xu View author publications You


can also search for this author inPubMed Google Scholar * I Irony View author publications You can also search for this author inPubMed Google Scholar * W W Bryan View author publications


You can also search for this author inPubMed Google Scholar * B Dunn View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence


to W W Bryan. ETHICS DECLARATIONS COMPETING INTERESTS The authors declare no conflict of interest. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE 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 Download citation * Received: 13


March 2017 * Revised: 13 July 2017 * Accepted: 18 July 2017 * Published: 24 July 2017 * Issue Date: September 2017 * DOI: https://doi.org/10.1038/gt.2017.64 SHARE THIS ARTICLE Anyone you


share the following link with will be able to read this content: Get shareable link Sorry, a shareable link is not currently available for this article. Copy to clipboard Provided by the


Springer Nature SharedIt content-sharing initiative