Martins Alexander C, Oshiro Mariana Y, Albericio Fernando, de la Torre Beatriz G
School of Health Sciences, UAM, Universidade Anhembi-Morumbi, São Paulo 03101-001, Brazil.
Medical Information Department, Thermo Fisher Scientific, São Paulo 4542011, Brazil.
Biomedicines. 2024 Sep 2;12(9):1992. doi: 10.3390/biomedicines12091992.
An increase in total drug (small molecules and biologics) approvals by the Food and Drug Administration (FDA) was seen in 2023 compared with the previous year. Cancer remained the disease most targeted by monoclonal antibodies (mAbs), followed by autoimmune conditions. Our data reveal the prevalence of approvals for biologics even during years when the total number of authorizations was low, such as in 2022. Over half the drugs that received the green light in 2023 benefited from expedited programs, as the incidence of many diseases increased. In addition, over half of the biologics approved received Orphan Drug Designation from the FDA. This narrative review delves into details of the most significant approvals in 2023, including mAbs, enzymes, and proteins, explaining their mechanisms of action, differences from previous drugs, placebo, and standards of care, and outcomes in clinical trials. Given the varying number of drugs authorized annually by the U.S. health authority, this review also examines the limits of external influences over the FDA's decisions and independence regarding drug approvals and withdrawals.
与前一年相比，美国食品药品监督管理局（FDA）在2023年批准的药物（小分子药物和生物制剂）总数有所增加。癌症仍然是单克隆抗体（mAb）最主要的靶向疾病，其次是自身免疫性疾病。我们的数据显示，即使在授权总数较低的年份（如2022年），生物制剂的获批率依然很高。由于许多疾病的发病率上升，2023年超过半数获批的药物受益于加速审批程序。此外，获批的生物制剂中有超过半数获得了FDA的孤儿药认定。这篇叙述性综述深入探讨了2023年最重要的获批药物的细节，包括单克隆抗体、酶和蛋白质，解释了它们的作用机制、与先前药物的差异、安慰剂以及治疗标准，还有临床试验结果。鉴于美国卫生当局每年批准的药物数量各不相同，本综述还探讨了外部因素对FDA药物审批和撤市决策及独立性的影响限度。
