The FDA would only withdraw a rare disease gene therapy for “pretty clear” reasons, such as when there is minimal benefit with extensive side effects. The one-and-done nature of administration also makes withdrawal easier, CBER Director Peter Marks says.

As COVID-19 vaccine sales decline, the company is investing billions in a diverse oncology pipeline including antibody drug conjugates, immunotherapy and cancer vaccines

Advances in synthetic biology are moving biopharma closer to a world where treatments can be tailored while remaining cost-effective.

Seeking to clear up a ‘misconception,’ Oncology Center of Excellence officials say earlier endpoints, such as progression-free survival and overall response, continue to be useful in getting therapies to market quicker, but sponsors still should plan to systematically collect OS data to ensure there is no detriment to survival.