Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

UsernamePublicRestriction

Gene Therapy: US FDA Willing To Accept Single-Arm Trials For Approval, But Sponsors May Hesitate

Executive Summary

Data indicates a smaller percentage of studies intended for product registration or indication expansion are single-arm trials, compared to the proportion for all gene therapy trials.

You may also be interested in...



Gene Therapy: US FDA Labeling For Vertex’s Casgevy, Bluebird’s Lyfgenia Reflect Different Risks

Agency grants same-day approval to the first two gene therapies for sickle cell disease; bluebird’s lentiviral-based lovo-cel carries a boxed label warning on hematologic malignancies, while Vertex’s CRISPR-Cas9 exa-cel carries a warning and precaution about potential off-target effects. Only the Vertex product qualified for a rare pediatric disease priority review voucher.

Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance

Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

US FDA’s Marks Willing To Accept Gene Therapies Occasionally Not Confirming Benefit

CBER director does not want to hold back potentially beneficial products, but also warned that patients will have difficult treatment decisions to make since an immune response might prevent a patient from receiving another gene therapy using the same vector ever again.

Related Content

Topics

Related Companies

Latest Headlines
See All
UsernamePublicRestriction

Register

PS149555

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

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

Cancel