uniQure files Huntington’s disease therapy applications

• uniQure submitted a BLA to the FDA and an MAA to the U.K. MHRA for AMT-130 in Huntington’s disease.
• The company requested FDA priority review, which could shorten the review period if granted.
• The filings are supported by three-year Phase I/II data showing slowed disease progression versus an external control.

uniQure (NASDAQ:QURE) submitted a Biologics License Application to the U.S. FDA and a Marketing Authorisation Application to the U.K. MHRA for ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for Huntington’s disease.

The company requested priority review for the FDA application, which if granted would reduce the FDA review cycle to six months after completion of the agency’s 60-day filing review period.

The regulatory filings are supported by three-year data from the Phase I/II program, where AMT-130 demonstrated a slowing of disease progression compared with a propensity score-matched external control from the Enroll-HD natural history database, while uniQure plans to present four-year study data before the end of the third quarter.

uniQure stated that AMT-130 has received FDA Breakthrough Therapy, RMAT, and Fast Track designations.

uniQure develops gene therapies for severe genetic diseases, including neurological disorders such as Huntington’s disease.

AMT-130 is the first Huntington’s disease therapy candidate to receive FDA Breakthrough Therapy and RMAT designations, according to uniQure, while the company continues regulatory discussions and clinical follow-up activities.