Gene Therapies for Rare Diseases September 13, 2018 Rare Disease - - PowerPoint PPT Presentation

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Gene Therapies for Rare Diseases September 13, 2018 Rare Disease - - PowerPoint PPT Presentation

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Gene Therapies for Rare Diseases September 13, 2018 Rare Disease Scientific Workshop Everylife Foundation for Rare Diseases Washington, DC Wilson W. Bryan Office of Tissues and Advanced Therapies (OTAT) Center for Biologics Evaluation and

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Gene Therapies for Rare Diseases

September 13, 2018 Rare Disease Scientific Workshop Everylife Foundation for Rare Diseases Washington, DC

Wilson W. Bryan Office of Tissues and Advanced Therapies (OTAT) Center for Biologics Evaluation and Research (CBER) United States Food and Drug Administration (US FDA)

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Center for Biologics Evaluation and Research (CBER)

Office of the Director Peter Marks, MD, PhD, Director Celia Witten, PhD, MD, Deputy Director Office of Vaccines Research and Review Office of Blood Research and Review

Office of Tissues and Advanced Therapies

Office of Management Office of Communication, Outreach, and Development Office of Compliance and Biologics Quality Office of Biostatistics and Epidemiology

www.fda.gov

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Cell and Gene Therapy

Investigational New Drug Applications

www.fda.gov

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Investigation

  • nal N

New Drug Application

  • ns (INDs)

for

  • r Gene Th

Therapy P Prod

  • duct

cts, 200 2002-2017 2017

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Gene Therapy: Scientific Advances

  • Human Genome Project
  • Completed in October 2003
  • 99% of human genes sequenced to 99% accuracy
  • Development of new vectors
  • Adeno-associated virus (AAV)
  • Lentivirus
  • Genome editing

www.fda.gov

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CAR T T Cells: A Novel el W Way t to T Treat Cancer er

Shannon L. Maude et al. Blood 2015 CTL, cytotoxic T lymphocyte; MHC, major histocompatibility complex

www.fda.gov

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Gene T Therapy United States M Marketing A g Approvals in 2017

  • Kymriah (tisagenlecleucel; Novartis)
  • CAR T cells (target – CD19)
  • Refractory childhood lymphoblastic B cell leukemia
  • Yescarta (axicabtagene ciloleucel; Gilead (Kite))
  • CAR T cells (target – CD19)
  • Adult patients with relapsed or refractory large B cell

lymphoma

  • Luxturna (voretigene neparvovec-rzyl; Spark

Therapeutics)

  • Gene therapy (adeno-associated virus vector)
  • Biallelic RPE65 mutation-associated retinal dystrophy

www.fda.gov

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www.fda.gov

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Baseball

www.fda.gov

Clinical Trials Preclinical

Marketing Application Post-marketing

BLA Phase 3 Phase 2 Phase 1 Preclinical Development

Post marketing

Drug Development

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www.fda.gov

Lou Gehrig Hank Aaron Babe Ruth

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Gene Therapies for Rare Diseases

FDA Draft Guidance (July 2018): Human Gene Therapy for Rare Disease

… consider designing … first-in-human study to be an adequate and well-controlled investigation that has the potential … to provide evidence of effectiveness to support a marketing application.

www.fda.gov

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Efficient Development of Gene Therapies 1) Teamwork Requires collaboration of scientists, academic investigators, sponsors (e.g., pharmaceutical industry), funding

  • rganizations, patients, patient

advocacy groups, and regulatory agencies

www.fda.gov

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Efficient Development of Gene Therapies 2) Preparation

  • Design and conduct Natural History studies

that will support subsequent drug development

  • When preclinical studies are beginning, draft

the design of the clinical study(ies)

  • Resolve manufacturing issues, as much as

possible, before first-in-human clinical trial

www.fda.gov

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Efficient Development of Gene Therapies 3) Try to hit a home run! Design first-in-human clinical trial to provide evidence of effectiveness (e.g., include randomized controls)

www.fda.gov

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Contact Information

  • Regulatory Questions:

OTAT Main Line – 240 402 8190 Email: OTATRPMS@fda.hhs.gov and Lori.Tull@fda.hhs.gov

  • OTAT Learn Webinar Series:

http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm

  • CBER website: www.fda.gov/BiologicsBloodVaccines/default.htm
  • Phone: 1-800-835-4709 or 240-402-8010
  • Consumer Affairs Branch: ocod@fda.hhs.gov
  • Manufacturers Assistance and Technical Training Branch: industry.biologics@fda.hhs.gov
  • Follow us on Twitter: https://www.twitter.com/fdacber

FDA Headquarters

www.fda.gov

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Acknowledgements

  • Rachael Anatol, PhD
  • Kimberly Benton, PhD
  • Larissa Lapteva, MD, MHS

www.fda.gov

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Wilson W. Bryan, M.D.

wilson.bryan@fda.hhs.gov

www.fda.gov

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Gene Therapy: Efficient Drug Development Drug development is like baseball: 1) Preparation 2) Teamwork 3) The heroes try for the home run!

www.fda.gov

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Baseball

www.fda.gov

Clinical Trials Preclinical

Marketing Application Post-marketing

BLA Phase 3 Phase 2 Phase 1 Preclinical Development

Post marketing

Drug Development

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Gene Therapies for Rare Diseases

September 13, 2018 Rare Disease Scientific Workshop Everylife Foundation for Rare Diseases Washington, DC

Wilson W. Bryan Office of Tissues and Advanced Therapies (OTAT) Center for Biologics Evaluation and Research (CBER) United States Food and Drug Administration (US FDA)