UniQure's Gene Therapy Faces FDA Challenges
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Mar 05 2026
0mins
Source: CNBC
- FDA Study Requirement: The U.S. FDA has mandated that UniQure conduct a placebo-controlled trial to validate the efficacy of its gene therapy for Huntington's disease, despite the company's claims that such a trial is unethical due to the need for prolonged general anesthesia.
- Clinical Trial Controversy: An FDA official indicated that UniQure's clinical trial data does not meet approval criteria, suggesting that the company may be aware of its earlier trial failures and has not pursued necessary clinical studies.
- Stock Price Fluctuation: Despite facing challenges from the FDA, UniQure's stock rose over 10% on Thursday, although it has fallen 58% this year, reflecting market concerns regarding the therapy's prospects.
- Regulatory Tensions: The relationship between UniQure and the FDA is increasingly strained, with the agency facing criticism for recent drug application rejections, and UniQure accusing the FDA of reversing its acceptance standards for the company's clinical trial data.
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Analyst Views on IONS
Wall Street analysts forecast IONS stock price to rise
14 Analyst Rating
13 Buy
1 Hold
0 Sell
Strong Buy
Current: 77.330
Low
65.00
Averages
92.67
High
110.00
Current: 77.330
Low
65.00
Averages
92.67
High
110.00
About IONS
Ionis Pharmaceuticals, Inc. develops six marketed medicines for serious diseases, including medicines for neurologic and cardiovascular diseases. Its marketed medicines consist of TRYNGOLZA, WAINUA (eplontersen), SPINRAZA (nusinersen), QALSODY (tofersen), TEGSEDI (inotersen) and WAYLIVRA (volanesorsen). TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS). WAINUA is approved for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults. SPINRAZA is used for the treatment of pediatric and adult patients with spinal muscular atrophy. QALSODY is approved for the treatment of Amyotrophic Lateral Sclerosis in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene, or SOD1-ALS. TEGSEDI is approved for the treatment of ATTRv-PN in adults. WAYLIVRA is approved as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk of acute, potentially fatal pancreatitis.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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