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UniQure needs to conduct another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said in a call with reporters on Thursday.
The official, who spoke on condition of anonymity before discussing confidential information, confirmed that the agency had asked the company to conduct a placebo-controlled trial of its treatment, which is administered directly into the brain. UniQure has said this type of study would not be ethical because it would require hours of general anesthesia, a characterization the official disputed.
“So what’s really going on? UniQure is the latest company to develop a failed therapy for HD patients,” the official said. “They probably recognize or understand on a deeper level that their study failed years ago, and instead of doing the right thing and conducting the right clinical trial, UniQure is running a distorted or manipulated comparison in the mind of the FDA.”
The comments mark the latest development in a messy public dispute between UniQure and the FDA and as the agency comes under fire for a series of recent rejections of drug applications, including some in which companies have accused it of deviating from previous guidance. FDA Commissioner Marty Makary appeared to criticize UniQure’s gene therapy for Huntington’s disease in an interview with CNBC’s Becky Quick last week. Makary did not name UniQure but described his treatment.
UniQure then 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 worsen without treatment, a so-called external control. UniQure has said that it would not be feasible to conduct a truly randomized, double-blind, placebo-controlled trial, which is considered the gold standard, because it would be ethical to subject people to hours of sham 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’ll have to see the data when we get it.'”
UniQure did not immediately comment.
The company’s stock rose more than 10% on Thursday and has fallen 58% this year since Thursday afternoon.
