UniQure
Coverage of UniQure in the Nexus archive.
- STAT+: Pharmalittle: We’re reading about another FDA reversal, pharma’s M&A spree and much more
The FDA will reconsider approving a gene therapy for a rare childhood brain disorder it previously rejected, following recent leadership changes. The U.S. also launched a trade investigation into Germany's plan to reduce pharmaceutical spending, which faced industry opposition and was revised.
- STAT+: FDA reverses course on Regenxbio’s childhood gene therapy after rejection
The FDA will reconsider approving Regenxbio’s gene therapy for a rare childhood brain disorder, mucopolysaccharidosis type II (MPS II), after initially rejecting it four months ago. This follows recent FDA reversals, including allowing UniQure to resubmit a gene therapy application for Huntington’s disease previously rejected by former commissioner Marty Makary.
- STAT+: Pharmalittle: We’re reading about hackers extorting Novo, an FDA about-face, and more
A cyber extortion group claimed to have stolen over a terabyte of data from Novo Nordisk, including proprietary information and AI model data, and demanded $25 million. The U.S. Food and Drug Administration reversed its opposition to an experimental Huntington’s disease treatment, allowing UniQure to pursue marketing approval.
- STAT+: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval
The FDA has reversed its opposition to UniQure's experimental Huntington’s disease treatment AMT-130, allowing the company to file for U.S. approval. UniQure plans to submit a marketing application in Q3 for accelerated approval, citing a three-year analysis of early-stage study data as acceptable to the FDA, despite previous objections from former agency officials.