Description: This combined session will take a deep dive into the importance of New Active Substance (NAS) status for the development of innovative therapies as a key criterion in the general regime of data and marketing protection for new medicines. Since the implementation of the NAS status in 2011, two guidelines for small molecules have been released, however, drug developers have awaited additional guidance from European Medicines Agency (EMA) on the criteria used for the assessment of NAS for biological substances. In November 2022, EMA published the first version of a reflection paper for the evaluation of NAS for biological substances including specific sections on Advanced Therapy Medicinal Products (ATMPs).
For the development of ATMPs or cell and gene therapies (C>s), the efforts of several stakeholders, including chemistry, manufacturing, and controls (CMC), nonclinical, clinical, regulatory, and commercial experts are required. On the way to market, pharmaceutical companies meet several scientific, regulatory, and operational challenges, which can lead to hinder the overall progress. The important steps for identifying development CMC challenges and strategies to efficiently resolve those that increase the likelihood of an expedited approval and delivering a new and approved (C>s) to patients in need are key to meet the end goals.
Learning Objectives:
The NAS status assessment, linked to the European Marketing Authorization, and to the definition of data and marketing protections.
Key concepts in the EMA’s reflection paper, outstanding scientific considerations, and potential impact on drug development strategies resulting from NAS criteria.
The available guidance related to the assessment focusing on biological products including ATMPs.
The key CMC challenges for the development of a (C>s).
The latest CMC strategies to overcome the challenges.
Apply the progressive regulatory requirements for the development of a (C>s).
