FDA official discusses UniQure gene therapy for Huntington’s disease

UniQure The company needs to conduct another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior FDA official said on a call with reporters Thursday.

The official, who requested anonymity before discussing sensitive information, confirmed that the agency had asked the company to conduct a controlled trial of the placebo, which is administered directly to the brain. UniQure said this type of study is unethical because it requires placing people under general anesthesia for hours, a characterization the official objected to.

“So, what’s really going on? UniQure is the latest company to provide a failed treatment for Huntington’s patients,” the official said. “They likely acknowledge or understand on a deep level that their trial failed years ago, and instead of doing the right thing and conducting the right clinical study, UniQure is making a distorted or manipulated comparison in the FDA’s mind.”

The comments represent the latest development in a messy public spat between UniQure and the FDA, with the agency under fire over a number of recent denials of drug approval applications, including some in which the companies accused it of reneging on previous guidance. FDA Commissioner Marty McCurry appeared to criticize UniQure’s gene therapy for Huntington’s disease in an interview with CNBC’s Becky Quick last week. McCary did not name UniQure but described his treatment.

UniQure then accused the FDA of reversing its position that the company’s clinical trial data would be sufficient to gain approval. The UniQure study used an external database to measure how patients with Huntington’s disease regressed without treatment, which is known as external control. UniQure said it would not be possible to conduct a true randomized, double-blind, placebo-controlled study, which is considered the gold standard, because it would not be ethical to make people undergo hours-long sham brain surgery.

The FDA official said the agency “never agreed to accept this distorted comparison” and that the FDA “never made 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 was down 58% this year as of Thursday afternoon.