QURE Submits Application for Gene Therapy for Huntington's Disease

uniQure N.V. QURE announced that it has submitted a Biologics License Application (“BLA”) to the FDA seeking accelerated approval for ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington’s disease.

The company has also requested Priority Review for the BLA. If granted, the designation would reduce the FDA’s review timeline to six months following the agency’s standard 60-day filing review period, potentially accelerating the regulatory path for AMT-130.

In parallel, uniQure submitted a Marketing Authorization Application (“MAA”) for AMT-130 to the United Kingdom’s Medicines and Healthcare products Regulatory Agency, marking another important regulatory milestone for the therapy.

The stock initially gained on the news, but the same was pared by the end of the day. Shares were down 3.41% yesterday.

Year to date, QURE stock surged 93.2% compared with the industry’s growth of 9.7%.

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More on QURE’s AMT-130

The BLA and MAA are supported by previously announced three-year data from the phase I/II clinical study of ifezuntirgene inilparvovec, which were compared with a propensity score-matched external control group derived from the Enroll-HD natural history database.

uniQure is conducting two multi-center phase I/II clinical studies evaluating the safety, tolerability and efficacy of ifezuntirgene inilparvovec for the treatment of Huntington’s disease.

The company plans to present four-year data from the ongoing phase I/II clinical studies before the end of the current quarter, providing additional insight into the therapy’s durability and potential clinical benefit.

Ifezuntirgene inilparvovec is the first investigational Huntington’s disease therapy to receive both Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the FDA.

The investigational gene therapy has also received Fast Track designation from the regulatory body, underscoring its potential to address an important unmet medical need in Huntington’s disease.

QURE utilizes a proprietary, gene-silencing miQURE platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

QURE’s Regulatory Submission Marks a Key Step Forward

The latest update relieves anxious investors as QURE had earlier suffered a regulatory setback. Shares crashed in March 2026 when the company announced that it had received final meeting minutes from the FDA regarding a type A meeting held in January 2026 to discuss AMT-130.

This meeting was held following an October 2025 pre-BLA meeting.

After receiving the final meeting minutes, the company disclosed that the FDA did not agree that data from the phase I/II studies, when compared with an external control group, were sufficient to serve as the primary evidence of effectiveness needed to support a marketing application for AMT-130.

The FDA then recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study.

A potential approval of this gene therapy will be a significant boost for QURE.

Huntington’s disease is a rare, inherited neurodegenerative disorder characterized by progressive motor symptoms, including chorea, as well as behavioral changes and cognitive decline, ultimately leading to worsening physical and mental function.

QURE's Zacks Rank & Stocks to Consider

uniQure currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the drug/biotech sector are Repligen RGEN and Anika Therapeutics ANIK, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while those for 2027 have increased from $2.57 to $2.61 during the same period. RGEN’s shares have gained 5.6% year to date.

Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.

Over the past 60 days, estimates for Anika Therapeutics’ 2026 EPS have risen from 41 cents to $1.05, while those for 2027 have increased from 46 cents to 95 cents over the same period. ANIK’s shares have surged 124.2% year to date.

 

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