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.

The Sweden-headquartered biotech has emphasized the promising survival data in a head and neck cancer study of setanaxib rather than the NOX inhibitor’s inability to shrink tumors.

Gestational age and a binary, neonatal composite endpoint are insufficient to assess outcomes that are important to babies and families, clinicians and researchers say, suggesting development of continuous variables or scales with weighted components and a comparison of outcomes among newborns in gestational age ‘buckets.’

The rumor mill is heating up on Stada’s potential sale by its owners, Bain Capital and Cinven, after seven years at the helm.