UniQure NV’s gene therapy for Huntington’s disease is a failed product, a senior US Food and Drug Administration official said Thursday, dimming the company’s hopes that it can persuade the agency to reconsider its demand for a rigorous new clinical trial.
In a call with reporters, the FDA official said the company needed to run a study that compared the fate of patients getting the therapy to those who underwent a sham surgery, since this type of a placebo control has been the agency’s standard when evaluating treatments for the rare, fatal condition. An earlier study was stone-cold negative, ...