On Wednesday, uniQure N.V. (NASDAQ:QURE) submitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for Huntington’s disease.
Regulatory Submissions For AMT-130
The company also submitted its marketing authorization application (MAA) for ifezuntirgene inilparvovec to the U.K. Medicines and Healthcare products Regulatory Agency (MHRA).
The BLA and MAA are supported by the previously announced three-year data analysis from the Phase 1/2 clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database.
The company intends to present a four-year data analysis from the ongoing Phase 1/2 studies before the end of the current third quarter.
Analyst Outlook And Sales Projections
On Wednesday, William Blair resumed coverage on uniQure with an Outperform rating and a $73 fair value estimate.
Analyst Sami Corwin sees a clearer pathway to BLA submission and approval for AMT-130.
“Given AMT-130’s tumultuous path to BLA submission, we see the BLA acceptance as an incremental catalyst for the stock, as it will further remove some regulatory risk, with potential approval being a substantial potential catalyst that could drive the stock toward SOTP value of $73,” analyst Corwin wrote on Wednesday.
William Blair also noted that the company is filing for approval in the UK in parallel, suggesting AMT-130 could be approved in two countries in 2027.
Although the launch will be measured given the requirement for surgically guided administration, analyst Corwin estimates peak global sales of $3.2 billion in 2035.
QURE Stock Price Activity: uniQure shares were down 4.95% at $45.49 on Wednesday, according to Benzinga Pro data.
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