The company intends to submit its formal application for the gene therapy in the third quarter of this year. While the three-year analysis serves as the primary support for this filing, the FDA has requested a collaborative framework for a subsequent confirmatory study to reinforce the clinical findings.

UniQure leadership stated they are prioritizing the design of this secondary study to ensure it aligns with agency expectations. By moving forward with the current data set, the company aims to expedite the regulatory process for a condition that currently lacks effective disease-modifying options.