Pink Sheet is part 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


Novartis' CAR-T Therapy Faces Quality, Safety Concerns At FDA Advisory Panel

Executive Summary

Unusual meeting agenda indicates that effectiveness of tisagenlecleucel-T in acute lymphoblastic leukemia is not a primary issue for US FDA advisory committee consideration at first-ever review of a CAR-T therapy; agency's draft questions focus on product quality considerations and measures for mitigating risk of cytokine release syndrome.

You may also be interested in...

Gene Therapies: Safety, CMC Draft Guidances Coming From US FDA In 2018

Lengthy safety registries, extensive premarket testing for replication competent retrovirus, and manufacturing submission advice likely; pending CBER documents informed by review and approval of Novartis' Kymriah, Kite's Yescarta, and Spark's Luxturna.

Novartis CAR-T AdComm Was Smooth Sailing, But Will That Be An Anomaly?

Other CAR-T sponsors can only hope for the same kind of US FDA advisory committee success that Novartis enjoyed with tisagenlecleucel, an outcome that reflected the sponsor's experience and preparation, a lack of major show-stopping safety or product quality concerns, and robust efficacy in a narrow pediatric population.

Novartis CAR-T Site Selection, Risk Management Are Model For Other Sponsors

US advisory committee's favorable views of Novartis' tisagenlecleucel-T suggest road map for other CAR-T developers to follow; with an Oct. 3 user fee deadline, pediatric leukemia agent is on path to becoming first chimeric antigen receptor T-cell therapy in US.

Related Content


Related Companies




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