UniQure says the U.S. Food and Drug Administration has reversed its prior stance on the company’s experimental Huntington’s disease gene therapy, allowing UniQure to move toward a U.S. approval application before running a new study. Multiple outlets report that the FDA previously challenged elements of the company’s submission plan following a public dispute involving clinical-trial data. After the reversal, UniQure can pursue an FDA pathway to seek approval without first completing an additional study, which the company frames as a change from the agency’s earlier position. The news comes after months of heightened scrutiny and disagreement between UniQure and FDA leadership about the evidence needed for review. Investors reacted strongly to the announcement, with at least one report describing a sharp jump in UniQure’s share price. The outlets characterize the development as a significant shift in the regulatory process for the therapy, though the exact timing and remaining requirements for approval are not detailed in the provided summaries. Overall, the reporting converges on the core point: the FDA is now permitting UniQure to submit the Huntington’s treatment for approval following its earlier opposition.
UniQure cleared to seek FDA approval for Huntington’s gene therapy after prior dispute
UniQure says the U.S. Food and Drug Administration has reversed its prior stance on the company’s experimental Huntington’s disease gene therapy, allowing UniQure to move toward a U.S. approval applic...
- UniQure announces the FDA has reversed its prior opposition to its Huntington’s disease gene therapy.
- The company says it is now cleared to submit an application for approval before conducting a new study.
- The change follows months of disagreement and public discussion between UniQure and FDA officials about clinical-trial data.
- At least one outlet reports UniQure shares rise sharply after the FDA reversal.
- The reports describe the development as a notable shift in the regulatory pathway for the therapy in the U.S.
The announcement comes months after UniQure became embroiled in a public debate with FDA leaders over the clinical trial data supporting its application.
2 months agoThe FDA has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its maker.
2 months agoUniQure NV shares soared after the company announced it will be able to seek US approval for its Huntington’s disease gene therapy before conducting a new study, a dramatic reversal that shows the Trump administration is relaxing its previous hard-line stance on drug reviews.
2 months ago
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