Cell, tissue and gene therapy (CGT) medicinal products / Advanced Therapy Medicinal Products (ATMPs) have made tremendous progress in the last decade. These products are considerably different from more conventional biotherapeutics such as monoclonal antibodies due to their inherent complex mechanism of action and variability. The process and product understanding for these molecules is limited/reduced, compared to more establish biologics. It is recognized that the standard biologics’ guidelines are not always applicable to these products and guidelines are being actively reviewed and updated by both the FDA, EMA and PDMA to ensure that companies have more relevant and applicable guidance to aid them during the drug development process. The development of these products is often against tight timelines and high cost, and many have complex but novel approaches to nonclinical and clinical programs. These products are often approved through expedited programs, which results in compression of the time available for CMC development.
Here we highlight the challenges encountered during the manufacturing of these products and the key regulatory support required to facilitate bringing these therapies to the market.
Manager, Regulatory Affairs CMC, PharmaLex
Anjali has a Ph.D. in Pharmaceutical Sciences with 7 years of drug development experience and over 3 years expertise in Regulatory Affairs (Authoring CMC modules for IND/IMPD/BLA/MAA submission, medical device dossier preparation, project management).
At PharmaLex, Anjali is a part of the CMC team and is accountable for the authoring of CMC modules, performing CMC gap analysis, providing solutions to meet regulatory requirements and supporting post-submission (MAA/BLA) activities as per EMA/FDA requests for biologics, biosimilars and Cell and Gene Therapy/ATMPs. Anjali also provides support for scientific advice briefing documents, and project management.