Gene therapy company UniQure has won a path toward FDA approval, after a regulatory rejection of its treatment was overturned in the wake of leadership changes at the agency.
According to Ars Technica, the FDA's earlier rejection of the gene therapy was overturned following the departure of Trump official Vinay Prasad. Ars Technica characterized the original rejection as "truly evil" in its reporting, and noted that UniQure secured another meeting with the FDA after Prasad's exit.
BioPharma Dive reports that UniQure now plans to file its gene therapy for approval. It frames the decision as a regulatory U-turn that followed the exits of two FDA figures, Marty Makary and Vinay Prasad. According to BioPharma Dive, some analysts read the reversal as a sign that current FDA leadership may be more flexible in certain cases.
The shift is notable because it ties a specific company's fortunes to who is sitting in key regulatory seats. A treatment that was blocked under one set of officials is now being waved toward a formal approval filing under another—without, in the sources provided, a stated change in the underlying science.
For patients waiting on gene therapies, and for investors trying to read the agency, the episode suggests that FDA decisions in this period may hinge partly on leadership turnover. Why it matters: when the same drug can be rejected and then welcomed depending on who runs the agency, it raises real questions about how predictable and consistent U.S. drug regulation is right now.