{"title":"Cell and Gene Therapy Products: Navigating the Regulatory Landscape of Paradigm Approvals in the US (2020 to 2024)","authors":"Ankita Arun Tandulje, Priya Changdev Varpe, Purva Dayanand Chaugule, Sneha Saji, Fathima Nizar, Rajeev Singh Raghuvanshi, Saurabh Srivastava","doi":"10.1007/s12247-025-09973-8","DOIUrl":null,"url":null,"abstract":"<div><h3>Purpose</h3><p>The purpose of this research is to analyze regulatory approvals of Cell and Gene Therapy Products by the FDA from 2020 to 2024. The study aims to explore the utilization of expedited regulatory pathways, including RMAT designation, and their impact on the approval trends of cell and gene therapies. Additionally, the research investigates therapeutic area-specific trends and highlights the significance of advanced Therapies in addressing unmet medical needs.</p><h3>Methods</h3><p>Data on FDA-approved Cell and Gene Therapies (2020–2024) were sourced from the FDA's public database. Approvals were analyzed by regulatory designations and therapeutic areas, with a focus on RMAT designation, introduced in 2016 to expedite advanced therapy approvals.</p><h3>Results</h3><p>Between 2020 and 2024, the FDA approved 25 Cell and Gene Therapy products, with 36% approved in 2024. The majority (34%) received Orphan Drug designation, followed by Breakthrough Therapy (20%) and RMAT (16%). Oncology led with 31% of approvals. RMAT requests and approvals increased, reaching a 70.7% approval rate in 2024, reflecting improved submission quality and regulatory alignment. These trends highlight the FDA's focus on innovation while maintaining safety and efficacy standards.</p><h3>Conclusion</h3><p>Between 2020 and 2024, 25 ATMPs were approved in the US, showcasing advancements in regenerative medicine, particularly in oncology and rare diseases. Expedited pathways like RMAT, Breakthrough Therapy, and Orphan Drug designations accelerated access while ensuring safety. These trends underscore the role of adaptive regulatory frameworks in driving innovation and addressing critical unmet needs, paving the way for future breakthroughs.</p><h3>Graphical Abstract</h3>\n<div><figure><div><div><picture><source><img></source></picture></div></div></figure></div></div>","PeriodicalId":656,"journal":{"name":"Journal of Pharmaceutical Innovation","volume":"20 2","pages":""},"PeriodicalIF":2.7000,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Pharmaceutical Innovation","FirstCategoryId":"3","ListUrlMain":"https://link.springer.com/article/10.1007/s12247-025-09973-8","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
Purpose
The purpose of this research is to analyze regulatory approvals of Cell and Gene Therapy Products by the FDA from 2020 to 2024. The study aims to explore the utilization of expedited regulatory pathways, including RMAT designation, and their impact on the approval trends of cell and gene therapies. Additionally, the research investigates therapeutic area-specific trends and highlights the significance of advanced Therapies in addressing unmet medical needs.
Methods
Data on FDA-approved Cell and Gene Therapies (2020–2024) were sourced from the FDA's public database. Approvals were analyzed by regulatory designations and therapeutic areas, with a focus on RMAT designation, introduced in 2016 to expedite advanced therapy approvals.
Results
Between 2020 and 2024, the FDA approved 25 Cell and Gene Therapy products, with 36% approved in 2024. The majority (34%) received Orphan Drug designation, followed by Breakthrough Therapy (20%) and RMAT (16%). Oncology led with 31% of approvals. RMAT requests and approvals increased, reaching a 70.7% approval rate in 2024, reflecting improved submission quality and regulatory alignment. These trends highlight the FDA's focus on innovation while maintaining safety and efficacy standards.
Conclusion
Between 2020 and 2024, 25 ATMPs were approved in the US, showcasing advancements in regenerative medicine, particularly in oncology and rare diseases. Expedited pathways like RMAT, Breakthrough Therapy, and Orphan Drug designations accelerated access while ensuring safety. These trends underscore the role of adaptive regulatory frameworks in driving innovation and addressing critical unmet needs, paving the way for future breakthroughs.
期刊介绍：
The Journal of Pharmaceutical Innovation (JPI), is an international, multidisciplinary peer-reviewed scientific journal dedicated to publishing high quality papers emphasizing innovative research and applied technologies within the pharmaceutical and biotechnology industries. JPI''s goal is to be the premier communication vehicle for the critical body of knowledge that is needed for scientific evolution and technical innovation, from R&D to market. Topics will fall under the following categories:
Materials science,
Product design,
Process design, optimization, automation and control,
Facilities; Information management,
Regulatory policy and strategy,
Supply chain developments ,
Education and professional development,
Journal of Pharmaceutical Innovation publishes four issues a year.
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