uniQure N.V. (NASDAQ:QURE) announced on Monday that the FDA has advised against using Phase 1/2 study data as primary evidence for the marketing application of AMT-130, its investigational gene therapy for Huntington’s disease.
The FDA strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study to proceed.
In November 2025, uniQure believed that the FDA currently disagrees that the data from the Phase 1/2 studies of AMT-130, in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission.
Next Steps
uniQure plans to request a Type B meeting in the second quarter of 2026 for further discussions.
“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said Matt Kapusta, CEO at uniQure. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130.”
FDA Rejection Signals Need For New Trials
uniQure’s setback comes as the company continues to advance its pipeline of proprietary gene therapies targeting severe diseases, including Huntington’s disease, refractory temporal lobe epilepsy, ALS, and Fabry disease.
In February, uniQure shared updated preliminary safety and exploratory efficacy data from 11 patients in its Phase 1/2a trial of AMT-191.
Dose-dependent elevations were observed across the three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above the mean normal level at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose.
The company’s previous success with gene therapy for hemophilia B highlights its capability in genomic medicine.
The company completed enrollment of the first cohort in the Phase 1/2a study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026.
uniQure reported cash, cash equivalents, and current investment securities of approximately $622.5 million as of December 31, 2025, expected to fund operations into the second half of 2029.
QURE Price Action: uniQure shares were down 43.38% at $8.85 during premarket trading on Monday. The stock is trading near its 52-week low of $7.76, according to Benzinga Pro data.
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