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UniQure needs to conduct another study to show that its gene therapy “really helps people with Huntington's disease,” a senior U.S. Food and Drug Administration official said in a call with reporters Thursday.
The official, who requested anonymity before discussing confidential information, confirmed that the agency asked the company to conduct a placebo-controlled trial of its treatment, which is administered directly to the brain. UniQure has said that type of study is unethical because it would require putting people under general anesthesia for hours, a characterization the official disputed.
“So what's really going on? UniQure is the latest company to make a failed therapy for Huntington's patients,” the official said. “They probably recognize or understand at some deep level that their trial failed years ago, and instead of doing the right thing and doing the right clinical study, UniQure is running a distorted or manipulated comparison in the FDA's mind.”
The comments mark the latest development in a confusing public dispute between UniQure and the FDA, and as the agency comes under fire for a series of recent rejections of drug approval applications, including some in which companies have accused it of flouting previous guidelines. FDA Commissioner Marty Makary, in an interview with CNBC's Becky Quick last week, appeared to criticize UniQure's gene therapy for Huntington's disease. Makary did not name UniQure but described his treatment.
UniQure later accused the FDA of changing its stance that the company's clinical trial data would be sufficient to seek approval. The UniQure study used an external database to measure how patients with Huntington's disease might get worse without treatment, known as external control. UniQure has said it would not be feasible to conduct a true randomized, double-blind, placebo-controlled study, considered the gold standard, because it would be unethical to have people undergo hours-long fake brain surgery.
The FDA official said the agency “never agreed to accept this distorted comparison” and the FDA “never makes such assurances.” Instead, “the FDA will always say, 'Well, we have to look at the data when we get it.'”
UniQure had no immediate comment.
The company's shares rose more than 10% on Thursday and have fallen 58% this year as of Thursday afternoon.
