Pink Sheet is part of the Business Intelligence Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


Hemophilia Gene Therapy Studies Could Use Non-Inferiority Design, US FDA Suggests

Executive Summary

Development guidance from CBER recommends comparing investigational gene therapy products to existing prophylaxis treatment.


Related Content

Historic Output Of Disease-Specific Guidances Is Among Gottlieb's Legacies At US FDA
Hemophilia Accelerated Approval: Factor Activity Useful Surrogate, Panel Says
Shutdown Bite Tightens At US FDA, But Gene Therapy To Get 50 More Reviewers
Bayer's Hemophilia A Portfolio Boosted by Jivi's EU Approval
Gene Therapy Guidance From US FDA Likely Needs Updating Sooner Rather Than Later
Hemophilia Seen As Good Testing Ground For Commercializing Gene Therapy
US FDA's Biologics Center Director Expects It Soon Will See 'Growth Spurt'
Hemlibra Hits The Heights In HAVEN Hemophilia Trials
Gottlieb On Gene Therapies: 'Very Seductive' To Think About Accelerated Approval Pathway
Roche's Hemlibra Priced And Labeled To Beat Competition, Safety Concern


Related Companies




Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts