CBER maps modernization plan to handle surge in research and applications.
FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.
Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.
The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.
These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.
These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.
In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].
Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.
Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.
Jill Wechsler is Washington editor for Pharmaceutical Technology.
Excerpt from:
FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine
- New gene therapy for sickle cell disease has been a long time coming, but is it a complete game-changer? - UCLA Health Connect - December 29th, 2023
- Sickle Cell Gene Therapy Reimbursement: Will Experience Matter? And Which Kind? - Pink Sheet - December 29th, 2023
- Cell and gene therapy will be top industry trend for pharma in 2024 - The Financial Express - December 29th, 2023
- Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy - AJMC.com Managed Markets Network - December 29th, 2023
- Global Cell and Gene Therapy Manufacturing Industry is projected to surpass a valuation of US$ 240 B - PharmiWeb.com - December 29th, 2023
- Penn's gene therapy layoffs are the latest in biotech belt-tightening in Philly and beyond - The Philadelphia Inquirer - December 29th, 2023
- Adeno-associated Virus Gene Therapy Market is Projected to Grow at a CAGR of 43.4% from 2023-2033 - EIN News - December 29th, 2023
- How Does Gene Therapy Work? Types, Uses, Safety - Healthline - May 9th, 2023
- Gene therapy: Comprehensive overview and therapeutic applications - April 7th, 2023
- Thanks to collaboration between Stand Up Therapeutics and VectorBuilder, a paraplegic patient will get gene therapy for the first time - Business... - January 21st, 2023
- Gene editing | Definition, History, & CRISPR-Cas9 | Britannica - January 4th, 2023
- A short history of gene therapy - Boston Children's Answers - December 27th, 2022
- Gene Therapy Analytical Development Summit 2022 | Home - December 27th, 2022
- $3.5-Million Hemophilia Gene Therapy Is World's Most Expensive Drug - Scientific American - December 10th, 2022
- CAR T Global Consultant Inc. Announce their Collaboration with Titronbio - a company founded in Shanghai China by a renowned leader in the field of... - December 10th, 2022
- Cell and Gene Therapy Manufacturing Services Market Size In 2023 | Financial Performance, In-Depth Insight of Trends, Key Players (Thermo Fisher... - December 10th, 2022
- How Gene Therapy Can Cure or Treat Diseases | FDA - December 2nd, 2022
- FDA Approves First Gene Therapy to Treat Adults with Hemophilia B - FDA.gov - November 24th, 2022
- Fact Check-mRNA vaccines are distinct from gene therapy ... - Reuters - October 29th, 2022
- Gene therapy: The Potential for Treating Type 1 Diabetes - Healthline - October 13th, 2022
- The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia - PMLiVE - October 13th, 2022
- Gene Therapy Hits Its Stride in the Clinic - Genetic Engineering & Biotechnology News - October 13th, 2022
- Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene... - October 13th, 2022
- Health Alert for Parents: How one boy is thriving following treatment with a gene therapy after receiving an early diagnosis - PR Newswire - October 13th, 2022
- M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress - Business Wire - October 13th, 2022
- Two Cell and Gene Therapies Manufactured at Lonza Houston Reach FDA Approval - Contract Pharma - October 13th, 2022
- Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive - October 13th, 2022
- Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market Report 2022 - ResearchAndMarkets.com - Business Wire - October 13th, 2022
- Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead - October 13th, 2022
- What Do We Know About Gene Therapy and Cystic Fibrosis? - The Epoch Times - October 13th, 2022
- CSafe and BioLife Solutions, Inc. announce partnership to expand supply chain solutions for the cell and gene therapy market - PR Newswire APAC - PR... - October 13th, 2022
- Genomics in Cancer Care Market is estimated to be US$ 72.61 billion by 2032 with a CAGR of 16.3% during the forecast period 2032 - By PMI -... - October 13th, 2022
- After $100M deal, J&J links gene therapy to improved vision in early-phase trial - FierceBiotech - October 4th, 2022
- Bayer Head Admits COVID-19 Vaccine is Gene Therapy - October 4th, 2022
- New Gene Therapy Shows Promise for Treating Age Related Macular ... - October 4th, 2022
- BioMarin resubmits its hemophilia gene therapy to the FDA - BioPharma Dive - October 4th, 2022
- Why Philly ranks #2 among best cell and gene therapy hubs in the US - Technical.ly - October 4th, 2022
- AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire - October 4th, 2022
- bluebird's (BLUE) Gene Therapy Approvals to Drive the Top Line - Zacks Investment Research - October 4th, 2022
- Researchers Develop Potential Gene Therapy to Treat Blindness - Managed Healthcare Executive - October 4th, 2022
- Voyager Therapeutics Announces Data Presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy - GlobeNewswire - October 4th, 2022
- Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application... - October 4th, 2022
- Sanofi partners with Scribe to gain gene editing tools for cell therapy work - BioPharma Dive - October 4th, 2022
- Real Endpoints Marketplace announces collaboration with bluebird bio to help scale delivery of a first-of-its-kind value-based contract for one-time... - October 4th, 2022
- Vertex given green light to seek US approval of CRISPR-based therapy - BioPharma Dive - October 4th, 2022
- Atsena Therapeutics Announces Positive Results from Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital... - October 4th, 2022
- Meet with the OrganaBio executives in-person at The Cell & Gene Meeting on the Mesa - Business Wire - October 4th, 2022
- Ocular Gene Therapy: A Literature Review With Focus on Current Clinical Trials - Cureus - September 25th, 2022
- Will experience support use of first-ever retinal gene therapy? - Ophthalmology Times - September 25th, 2022
- Rejuvenate Bio CEO highlights ambitious approach of using gene therapy to reverse aging - MedCity News - September 25th, 2022
- Gene therapy R&D market is projected to grow at a CAGR of 30.1% by 2032: Visiongain Reports Ltd - GlobeNewswire - September 25th, 2022
- Emulate Announces New Gene Therapy Application Enabling Accelerated Development of Treatments for Genetic Diseases with Organ-on-a-Chip Technology -... - September 25th, 2022
- CSL flexes gene therapy muscle with latest drug - Sydney Morning Herald - September 25th, 2022
- FDA Updates for the Week of Sept. 19, 2022 - Managed Healthcare Executive - September 25th, 2022
- AVROBIO Receives Rare Pediatric Disease Designation From FDA For First Gene Therapy In Development For Cy - Benzinga - September 25th, 2022
- Preparing Europe for a new generation of innovative therapies - Open Access Government - September 25th, 2022
- KSQ Therapeutics Announces Leadership and Board Additions - Business Wire - September 25th, 2022
- Hemophilia gene therapies from BioMarin, CSL pick up an early ICER endorsement - FiercePharma - September 16th, 2022
- Challenges In Gene Therapy - University of Utah - September 16th, 2022
- Hopkinton bioscience company forms advisory board for cell and gene therapy - Worcester Business Journal - September 16th, 2022
- Gene Therapy Restores Some Vision in Colorblind Children - Pharmacy Times - September 16th, 2022
- Coave Therapeutics partners with IMN on gene therapy - Labiotech.eu - September 16th, 2022
- MeiraGTx's Industry-Leading Gene Therapy Manufacturing Facility in Shannon, Ireland Formally Introduced by Head of Irish Government -... - September 16th, 2022
- Bluebird CFO resigns ahead of gene therapy launch - BioPharma Dive - September 16th, 2022
- Enrollment completed in STARLIGHT Phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease - Ophthalmology Times - September 16th, 2022
- Global Cancer Gene Therapy Market to Reach $11.35 Billion by 2030 at a CAGR of 23.3% - PR Newswire - September 16th, 2022
- How WhiteLab Genomics is using AI to aid gene and cell therapy development - TechCrunch - September 16th, 2022
- CAR-T Cell Therapy Market Segmentation and Forecast Analysis up to 2027 - BioSpace - September 16th, 2022
- Cluster-busters against Cancer and a Potential New Gene Therapy for LCA - BioSpace - September 16th, 2022
- Ori Biotech and CTMC team up on cell therapy delivery - BioPharma-Reporter.com - September 16th, 2022
- Adverum (ADVM) Doses First Patient in the Mid-Stage wAMD Study - Zacks Investment Research - September 16th, 2022
- Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today - September 8th, 2022
- A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review - September 8th, 2022
- Myrtelles rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European... - September 8th, 2022
- Sangamo presses ahead with Fabry disease gene therapy - BioPharma Dive - September 8th, 2022
- The gene therapy that could transform the lives of millions - ABC News - September 8th, 2022
- As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer - September 8th, 2022
- Mayflower BioVentures to Announce New Cell & Gene Therapy Companies within Months - BioSpace - September 8th, 2022
- Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire - September 8th, 2022
- AnGes : Announces Discontinuation of Development of HGF Gene Therapy Product for Additional Indication of Chronic Arterial Occlusive Disease with Rest... - September 8th, 2022