On February 26 2026, uniQure N.V. shares fell sharply after comments from FDA Commissioner Marty Makary were interpreted as a negative signal for the company’s Huntington’s disease gene‑therapy candidate, AMT‑130. The remarks came amid ongoing uncertainty over the FDA’s stance on the external‑control data used in AMT‑130’s pivotal study, a key issue that has already delayed the company’s BLA timeline. Investors reacted to the perception that the regulator’s comments could indicate a further shift away from the data strategy that uniQure has built its approval case around.
AMT‑130 has been the centerpiece of uniQure’s pipeline, with Phase I/II data showing a 75 % slowing of disease progression at 36 months. The FDA’s recent feedback has cast doubt on whether the external‑control methodology will be accepted as primary evidence, potentially requiring a costly and time‑consuming confirmatory trial. UniQure’s cash position of $694.2 million, as of September 30 2025, provides a runway to 2029, but the regulatory uncertainty threatens the near‑term commercial prospects of its flagship therapy.
Management has expressed surprise and disappointment at the FDA’s shift. CEO Matt Kapusta said, “We are surprised by the FDA’s feedback at the recent pre‑BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural‑history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway.” He added that the company remains committed to working with the FDA to determine the best path forward to bring AMT‑130 to patients in the U.S. The comments highlight the centrality of AMT‑130 to uniQure’s valuation and the heightened risk profile associated with regulatory setbacks.
The market reaction underscores the sensitivity of uniQure’s share price to FDA commentary and the broader regulatory environment for gene therapies. Investors are closely monitoring the company’s next steps, as any further regulatory setbacks could erode confidence in AMT‑130’s approval prospects and impact uniQure’s valuation, which is heavily weighted on the success of this single product.
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