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.

After several rejections by NICE and an appeal by Pfizer, the company has reduced the price for its first-in-class sickle cell disease drug to a level that the health technology assessment institute deems acceptable.