uniQure files Huntington's gene therapy AMT-130 for U.S., U.K. approval

What happened

uniQure N.V. (NASDAQ: QURE), a Dutch gene therapy developer, has filed for regulatory approval of its experimental treatment for Huntington's disease, AMT-130, in both the United States and the United Kingdom. The company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration, according to NeurologyLive, and has also applied to UK regulators, as reported by Reuters.

The applications are based on previously announced three-year data showing AMT-130 could significantly slow signs of disease progression, according to BioPharma Dive. The company had earlier faced an FDA reversal, with Reuters noting the filing came "after FDA reversal."

Shares of uniQure closed at $44.50 on Monday, down 0.8% from the prior close of $44.86, according to price data. The stock has a 52-week range of $8.73 to $71.50 and a market cap of about $3.09 billion, per MarketBeat.

Why it matters

AMT-130 is a gene therapy designed to treat Huntington's disease, a rare, inherited neurodegenerative disorder. There is currently no approved gene therapy for Huntington's, so if approved, AMT-130 would be the first in this indication, a milestone that has drawn attention from Wall Street analysts and patient advocacy groups.

The filing advances a high-profile rare-disease asset toward possible approval and is a material catalyst for this mid-cap biotech, which has seen its stock rise over 20% recently following analyst upgrades citing an improving regulatory outlook, according to StockTwits.

The European Huntington Association posted on Facebook that the news was "what our community needed," linking to uniQure's press release.

What this means

A Biologics License Application (BLA) is the U.S. regulatory submission required to market a biologic product, such as a gene therapy, in the United States. It is analogous to a New Drug Application for small-molecule drugs. Filing a BLA is a formal request for FDA approval to sell the product commercially.

AMT-130 is an experimental gene therapy that uses a viral vector to deliver a gene designed to reduce production of the toxic protein that causes Huntington's disease. In the U.S. and U.K., regulators will now review the safety and efficacy data from clinical trials to decide whether the benefits outweigh the risks.

Normally, after a BLA is filed, the FDA has 60 days to decide whether to accept it for review, and then a review process follows that may involve an advisory committee and a final decision. The timeline can vary, but the filing itself is a required step toward any potential approval.

The company's prior "FDA reversal" suggests the path here has not been straightforward. Reuters did not detail the nature of that reversal, but such events can involve clinical holds or other regulatory setbacks that delay or complicate a filing. The fact that uniQure has submitted despite that history indicates the company believes its data are now sufficient.

It is not yet known whether regulators will accept the applications or how long their reviews will take. As with any experimental therapy, approval is not guaranteed.