UniQure announced on Monday that its efforts to secure approval for a Huntington’s disease treatment from the Food and Drug Administration (FDA) remain obstructed, marking another hurdle for a therapy that previously seemed poised for approval.
In a recent meeting with the FDA, UniQure learned that regulators remain unconvinced that the data from its completed single-arm clinical trial of the gene therapy, AMT-130, are adequate for a marketing application. The FDA has indicated that a more rigorous study design is necessary.
The agency has recommended that UniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study to strengthen its application. This requirement underscores the FDA’s commitment to ensuring robust evidence of efficacy and safety, which may significantly delay the potential market entry of AMT-130 and impact UniQure’s strategic positioning in the competitive landscape of gene therapies.
Use the database as your supply chain compass →
