The European Medicines Agency (EMA) set aside a whole new category for such products under the Advanced Therapy Medicinal Products (ATMP) category, which encompasses medicines for human use that are based on genes, tissues or cells that, as the EMA says, “offer groundbreaking new opportunities for the treatment of disease and injury.”

 

With its assessment that there aren’t enough CMOs yet to meet demand, GlobalData sees a problem ahead, as this disconnect will become very apparent when more and more ATMPs receive regulatory approval. The lack of enough CMOs will potentially create bottlenecks to large-scale commercial manufacture.

 

Currently, 79 of these potential cell and gene therapies are for COVID-19, though the majority of these are in preclinical and discovery phases. Two of them are in Phase 3 trials—Mesoblast’s remestemcel-L and Athersys’s MultiStem—with another 15 in Phase 2 trials. If these are approved, they will need to be scaled up and manufactured quickly to treat the current pandemic, GlobalData notes.

 

“There are considerable opportunities for CMOs with the capability to manufacture ATMPs. However, only 152 CMOs have the capability to produce cell or gene therapy APIs for global markets, and of these only 121 are dedicated CMOs,” said Adam Bradbury, a PharmSource analyst at GlobalData. “Producing gene or cell therapies requires an inherently high level of manufacturing expertise and expensive facility requirements that many pharma companies do not possess.”

 

Concluded Bradbury: “With a large number of ATMPs in the drug pipeline greater numbers will be approved. This will require improvements to the production process and the removal of manufacturing bottlenecks to create cost-efficient manufacture at a commercial scale.”