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Regulatory News | 05 January 2018 | By Michael Mezher
In his second annual report as the director of the US Food Administration's (FDA) Center for Biologics Research and Evaluation(CBER), Peter Marks said the center continued to meet or exceed its performance goals in the final year of the last Prescription Drug User Fee Act (PDUFA V).
According to Marks, CBER exceeded its PDUFA goals by completing 100% of its FY2017 cohort of standard and priority biologics license application (BLA) and new drug application (NDA) reviews two months ahead of schedule.
The center also acted on all standard and priority efficacy supplements that were due in FY2017, and exceeded its target for reviewing 510(k) submissions by coming to a decision on all 510(k)s within 90 days.
Going beyond CBER's performance metrics, Marks says he is "particularly proud to have approved several therapeutic 'firsts'" in 2017.
Perhaps the most significant of which is the first US approval of a gene therapy, Novartis' chimeric antigen receptor T cell (CAR-T) therapy Kymriah (tisagenlecleucel) in August. The approval, which was dubbed an "historic action" by the agency was quickly followed by the approval of a second CAR-T therapy in October, Gilead's Yescarta (axicabtagene ciloleucel).
CBER also approved several other therapeutic firsts in FY2017, including MACI, an autologous cellularized scaffold for repairing knee cartilage; Odactra, the first allergen extract that can be taken sublingually; and Haegarda, the first subcutaneous C1 esterase inhibitor for preventing hereditary angioedema (HAE) attacks.
Marks further touted CBER's work on international public health issues in 2017, including completing seven expedited reviews for investigational new drug (IND) applications for Zika vaccines and providing assistance to other regulators on safety and efficacy concerns for investigational Ebola vaccines.
In addition, CBER issued or contributed to a number of significant guidance documents in FY2017, including guidances on demonstrating biosimilar interchangeability, statistical approaches to evaluate analytical similarity, deviation reporting for human cells, tissues and cellular and tissue-based products and guidance related to blood and tissue donor screening.
Tags: PDUFA, CBER, Kymriah