Drug Product Technology and Engineering, Amgen, Cambridge, Massachusetts, USA.
Pharmaceutical Development, Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.
Nucleic Acid Ther. 2023 Jun;33(3):159-177. doi: 10.1089/nat.2022.0073. Epub 2023 Feb 14.
A primary function of the parenteral drug product manufacturing process is to ensure sterility of the final product. The two most common methods for sterilizing parenteral drug products are terminal sterilization (TS), whereby the drug product is sterilized in the final container following filling and finish, and membrane sterilization, whereby the product stream is sterilized by membrane filtration and filled into presterilized containers in an aseptic processing environment. Although TS provides greater sterility assurance than membrane sterilization and aseptic processing, not all drug products are amenable to TS processes, which typically involve heat treatment or exposure to ionizing radiation. Oligonucleotides represent an emerging class of therapeutics with great potential for treating a broad range of indications, including previously undruggable targets. Owing to their size, structural complexity, and relative lack of governing regulations, several challenges in drug development are unique to oligonucleotides. This exceptionality justifies a focused assessment of traditional chemistry, manufacturing, and control strategies before their adoption. In this article, we review the current state of sterile oligonucleotide drug product processing, highlight the key aspects to consider when assessing options for product sterilization, and provide recommendations to aid in the successful evaluation and development of TS processes. We also explore current regulatory expectations and provide our interpretation as it pertains to oligonucleotide drug products.
注射剂药品制造工艺的主要功能之一是确保最终产品的无菌性。对注射剂药品进行灭菌的两种最常见方法是终端灭菌(TS)和膜过滤除菌,前者是在灌装和完成后对最终容器内的药品进行灭菌,后者是通过膜过滤对产品流进行灭菌,然后在无菌处理环境中将其灌装到预先灭菌的容器中。虽然 TS 比膜过滤除菌和无菌处理提供了更大的无菌保证,但并非所有药品都适用于 TS 工艺,因为 TS 工艺通常涉及热处理或电离辐射。寡核苷酸是一类新兴的治疗药物,具有治疗广泛适应症的巨大潜力,包括以前无法治疗的靶点。由于其大小、结构复杂性以及相对缺乏监管法规,寡核苷酸药物开发中的几个挑战是独特的。这种特殊性证明在采用传统的化学、制造和控制策略之前,对其进行重点评估是合理的。本文综述了无菌寡核苷酸药品加工的现状,强调了在评估产品灭菌选择时需要考虑的关键方面,并提供了建议,以帮助成功评估和开发 TS 工艺。我们还探讨了当前的监管期望,并对其与寡核苷酸药品的关系进行了阐释。