The number of new drugs containing new active substances approved in the EU rose significantly in 2020, underpinned by a strong showing for orphan-designated products and a string of novel medicines for cancer and infectious diseases. Notable among the new clutch of approvals was the first ever COVID-19 vaccine, Pfizer/BioNTech's Comirnaty.

The development and commercialization of cell and gene therapies during a pandemic, with regulators and payers demanding more data on effect duration and safety, has resulted in headwinds. Added to manufacturing and clinical trial recruitment issues, investors’ patience has been strained.

Regulatory review resource-intensive cell and gene therapy licensing applications are rolling toward US FDA along expedited pathways, while complete response letters and missed goal dates start to stack up under pandemic inspection constraints.

Product review report reveals multiple delays and missteps on route to market, holding valuable lessons for other gene therapy developers but also evidencing an ultimately welcoming environment.