Artikel
Mitigating regulatory risk with holistic CMC strategies
This article was originally published on European Biotechnology
As advanced therapeutic medicinal products (ATMPs) and other next-generation biologics move toward approval, Chemistry, Manufacturing, and Controls (CMC) continues to be the most frequent source of regulatory concern. This article highlights recurring CMC barriers and best practices to mitigate approval delays or refusals.
As more advanced therapeutic medicinal products (ATMPs) and other next-generation biologics seek regulatory approval, the development challenges these products encounter is gaining prominence. Assessments from regulatory authorities consistently show that Chemistry, Manufacturing and Controls (CMC) remains the area of greatest concern.
Last year, the US Food and Drug Administration (FDA) published more than 200 complete response letters (CRLs) that had been issued between 2002 and 2024 for products that have since been approved, providing greater transparency into the agency’s decision-making process. Additionally, FDA has rejected or delayed several new cell and gene therapy (CGT) biologics license application (BLA) submissions due to problems with manufacturing readiness.
As more advanced therapeutic medicinal products (ATMPs) and other next-generation biologics seek regulatory approval, the development challenges these products encounter is gaining prominence. Assessments from regulatory authorities consistently show that Chemistry, Manufacturing and Controls (CMC) remains the area of greatest concern.
Last year, the US Food and Drug Administration (FDA) published more than 200 complete response letters (CRLs) that had been issued between 2002 and 2024 for products that have since been approved, providing greater transparency into the agency’s decision-making process. Additionally, FDA has rejected or delayed several new cell and gene therapy (CGT) biologics license application (BLA) submissions due to problems with manufacturing readiness.
According to the manufacturers of these products, deficiencies included process control gaps, inadequate stability data, unvalidated analytical methods, and ongoing issues following Good Manufacturing Practice (GMP) inspections.
These CMC and GMP issues are well-documented in Europe, where most biological products and all ATMPs go through the Centralised Procedure (CP) with the European Medicines Agency (EMA). The agency has a long-established documentation framework and each product is subject to European public assessment reports (EPARs), whether approved or denied authorization. The release of the CRLs from FDA now allows observers to more rigorously assess areas of respective interest and concern between the health authorities.
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