UniQure N.V.’s (NASDAQ: QURE) stock is rising sharply this morning after the company announced a significant milestone in its regulatory licensing course. According to the most recent check, the price of QURE shares jumped 90.54% to $13.90.
uniQURE get The FDA’s Approval for AMT-130
The increase in shares is due to UniQure’s successful agreement with the U.S. Food and Drug Administration (FDA) on key components of the Accelerated Approval pathway for AMT-130, a possible therapy for Huntington’s disease.
The collaboration demonstrates the quality of uniQure’s data and the company’s successful partnership with the FDA’s Center for Biologics Evaluation and Research (CBER). The company’s attempts to provide a potentially life-changing medication to those with Huntington’s disease have reached a major turning point.
Fast-Tracking Treatment for Huntington’s Disease
The accord speeds up the availability of Accelerated Huntington’s Disease Therapy AMT-130 and allows UniQure to adopt a more direct regulatory strategy. This accomplishment has a lot of potential for the Huntington’s disease community, putting uniQure in a position to offer a novel treatment for an illness with few other alternatives.
In order to meet statistical analysis and technical production needs, the business has already started preparing for the Biologics License Application (BLA) filing and expects to have more conversations with the FDA in early 2025.
FDA Approves Key Data for BLA Submission
The FDA acknowledged during its Regenerative Medicine Advanced Therapy (RMAT) Type B conference that the BLA submission may be based primarily on data from the ongoing Phase I/II studies, obviating the necessity for a separate pre-submission study.
The FDA also supported the validity of AMT-130’s clinical profile by acknowledging the possibility of neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) as a supporting measure for treatment effectiveness. Following AMT-130’s RMAT designation in May 2024, this regulatory advancement underscores the drug’s potential to address major unmet medical needs in the treatment of Huntington’s disease.
