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Gene therapy gets FDA approval -- and a $2 million price tag. ... In January, FDA leaders said they expect that the agency will be approving 10 to 20 cell and gene therapy products yearly by 2025.


Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed ...


In August, the FDA approved Kymriah, the first gene-therapy product to treat a form of leukemia. A second gene-based treatment, called Yescarta, was approved for a form of lymphoma in October.


Currently, there are fewer than five approved gene therapies on the U.S. market. But a report by the Alliance for Regenerative Medicine showed that there were 372 gene therapy products in clinical trials in the first quarter of this year (a 17 percent year-over-year increase from 2018).


Zolgensma was then approved in 2019 to treat Spinal Muscular Atrophy. There are now many additional companies moving aggressively to develop their pipelines of gene therapy products. The specifics of each gene therapy is slightly different, however the main challenge is to replace a missing or non-functional protein with a functional protein.


The EMA withdrew the authorization for two tissue engineering products, one cell therapy and one gene therapy, and New Zealand lapsed approval of one cell therapy. Most products were first authorized after 2010, including 10 (83.3%) gene therapies, 13 (72.2%) cell therapies, and 13 (56.5%) tissue-engineered products.


Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while ...


FDA Commissioner Scott Gottlieb, M.D. and Director of the Center for Biologics Evaluation and Research (CBER) Peter Marks, M.D., Ph.D., released a joint statement on January 15, 2019 previewing new FDA policies to advance the development of safe and effective cell and gene therapies.. This statement comes amid expectations for record numbers of Investigational New Drug (IND) applications and ...


Gottlieb added, “Today’s approval marks another first in the field of gene therapy—both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss….The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious ...


CASSS Cell and Gene Therapy Workshop. Regulatory Updates from Across the Globe. July 11, 2018. Regulatory Updates for Human Gene Therapy Products: An FDA Perspective Denise Gavin, PhD. Chief, Gene Therapy Branch. Division of Cellular and Gene Therapies. Office of Tissues and Advanced Therapies. Center for Biologics Evaluation and Research