UniQure’s market value plunged after FDA Commissioner Marty Makary defended recent rare‑disease therapy rejections on a public broadcast, prompting investor concerns that the agency’s stance could complicate approvals for certain delivery approaches. UniQure shares fell more than 30% amid investor speculation that Makary’s remarks targeted therapies administered via burr‑hole delivery, a description some associated with UniQure’s AMT‑130 program. Separately, STAT reported Makary’s broader defense of the agency and of CBER leadership, underscoring a regulatory tone that has left several rare‑disease developers uncertain about approval pathways. Industry analysts stressed that the Commissioner’s comments, while not naming companies, amplified regulatory risk perceptions and may further complicate sponsor engagement with the FDA. Companies developing invasive or novel delivery modalities will likely increase dialog with regulators and hedge programs with additional data or alternative delivery strategies to mitigate approval uncertainty.