BioPharm International - March2020

BioPharm International - Regulatory

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8 BioPharm International eBook March 2020 Inside FDA's New Gene and Cell Therapy Guidance In January 2020, the agency finalized six clinical development and manufacturing guidance documents and drafted new guidance on what would qualify new gene therapies as orphan drugs. S ince 2017, FDA has approved four gene ther- apies, and regulators expect the number of gene and cell therapies under development to increase dramatically. FDA has already received more than 900 investigational new drug (IND) applications for clinical studies involving these therapies (1). FDA has published guidance on cell and gene thera- pies since 1998, when the field was brand new, according to Kevin Hacker, director of life sciences at Clarkston Consulting (2). To help spur innovation, FDA offers cell and gene ther- apy developers a number of expedited review options, including "breakthrough therapy" and "fast track." In 2018, the agency introduced another category, "regenera- tive medicine advanced therapy," and published six draft guidances on the following topics: • Gene therapies for hemophilia and retinal disor- ders, as well as for rare diseases in general • Testing retroviral vector-based gene therapy prod- ucts during manufacture and patient follow-up • Long-term patient follow-up and monitoring for adverse events after treatment with gene therapy • Chemistry, manufacturing, and control (CMC) information that should be included in investi- gational new drug applications (IND) for gene therapies. FDA updated and finalized these guidance documents on January 28, 2020, with the stated goal of support- ipopba - AGNES SHANLEY Regulatory Sourcebook Gene and Cell Therapies

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