FDA Shifts Remain Focus for Cell and Gene Therapy Industry at European Meeting

Cell and gene therapy makers met in Rome last week to discuss the sector's prospects in Europe. A main topic at the Alliance for Regenerative Medicine’s Meeting on the Med remained developments at the Food and Drug Administration more than 4,500 miles away.

The leader of an organization representing these manufacturers pointed to recent approvals of rare disease treatments by Rocket Pharmaceuticals and Regeneron Pharmaceuticals as reasons for optimism in the sector. He also noted the departure last week of the FDA’s top regulator of gene and cell therapies as well as vaccines.

The regulator’s exit was referenced as a potentially positive development for the field. The agency’s rejection rate for cell and gene therapies has doubled since 2024. The meeting was organized to focus on opportunities and challenges for the sector in Europe.

Discussions nonetheless returned to decisions made by the FDA.

Pharmaceuticals secured approvals for treatments targeting rare diseases. These approvals were cited as positive signals amid broader uncertainty in the field.

The FDA’s top regulator for gene and cell therapies left the agency last week. The rejection rate for these therapies has doubled since 2024 according to meeting participants.