Keep up with the latest regulatory news and topics here

BLA review updates

The big BLA news of the 4th quarter of 2023 was the approval of both bluebird bio’s Lyfgenia (lovo-cel) and Vertex’s Casgevy (exa-cel) for sickle cell disease on December 8, 2023. This brings the total of new BLA approvals for 2023 to seven.

To start out the 1st quarter of 2024, FDA approved the indication of transfusion-dependent beta thalassemia (TDT) for Vertex’s Casgevy (exa-cel) on January 16, 2024.

Brainstorm withdrew their BLA submission for debamestrocel for treatment of amyotrophic lateral sclerosis (ALS).  

BLA approvals in 2023

List from 2023 and duplicate for 2024

CGT BLAs currently under US FDA review

The following information on BLAs under US FDA review was obtained from company press releases.

Past and Upcoming FDA Town Halls

The following links to the Meeting Materials include access to the recorded video link, transcript, or slides. 
Nonclinical Assessment of Cell and Gene Therapy Products
August 30, 2023
Decentralized Clinical Trials
June 20, 2023
Regulatory Education for Industry (REdI) Annual Conference 2023
June 5-9, 2023
OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 2023
June 8, 2023
OTP Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls – April 2023
April 25, 2023
Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products
April 27, 2023
Clinical Trials: The Patient Experience
April 13, 2023
FDA CBER Office of Therapeutic Products (OTP) Advanced Manufacturing and Analytical Technologies (AMAT) for Regenerative Medicine Therapies (RMT) Workshop
March 14, 2023
OTAT Town Hall: Clinical Development of Gene Therapy Products for Rare Diseases
February 7, 2023
Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products
December 16, 2022
OTAT Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls
December 7, 2022
FDA/PQRI Workshop on the Regulatory Framework for Distributed and Point of Care Pharmaceutical Manufacturing: An Opportunity for DM/POC Stakeholder Engagement
November 14 - 16, 2022
FDA CBER OTAT Patient-Focused Drug Development Listening Meeting — Patient Perspectives on Gene Therapy Products
November 15, 2022
Natural History Studies to Support Regenerative Medicine: A How-To Webinar
October 27, 2022
OTAT Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls
September 29, 2022
2022 Center for Biologics Evaluation and Research (CBER) Science Symposium
September 20-22, 2022
Regulatory Education for Industry (REdI) Annual Conference 2022
June 6 - 10, 2022
Annual Patient Engagement & Regenerative Medicine Meeting 2022: An FDA CBER Workshop for Patient Advocates
May 24, 2022
RegenMedEd Webinar: The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases
March 9, 2022

Upcoming FDA AdComm meetings

No upcoming meetings of the Cellular, Tissue, and Gene Therapies Advisory committee have been announced.

A date for the Oncologic Drug Advisory Committee (ODAC) meeting to discuss data from BMS’s BLA supplement for approval of ABECMA for earlier line of treatment has not yet been announced.

Regulatory Roundup LIVE

Our first Regulatory Roundup LIVE was our Unbridled Excellence #4 and featured our unparalleled team of former FDA regulators discussing the latest and greatest regulatory news...that's DHC Master Principal Kimberly Benton and Master Practice Expert Don Fink, as well as Senior Principal Heath Coats, Principal Kevin Whittlesey, and Practice Expert Tal Salz. Go to the main Unbridled Excellence page to watch/download #4 on demand.