FDA Approves Therapy for Rare Disease Without Randomized Trial Data

‘Randomized trials are not always needed to approve medical products,’ Dr. Vinay Prasad said.

The U.S. Food and Drug Administration (FDA) in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

The Food and Drug Administration on Aug. 14 approved a therapy for a rare disease called recurrent respiratory papillomatosis (RRP).

Regulators cleared Papzimeos, an immunotherapy made by Precigen, citing data from a single-arm, open-label trial that looked at how it performed in adults with RRP and who needed at least three surgeries a year.