FDA official discusses UniQure gene therapy for Huntington’s disease
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UniQure Another study is needed to prove that gene therapy “really helps people with Huntington’s disease,” a senior official with the U.S. Food and Drug Administration 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 placebo-controlled trial for the treatment administered directly to the brain. UniQure said such a study was unethical because it would require keeping people under general anesthesia for hours, a characterization the official disputed.
“So what’s really going on? UniQure is the latest company to deliver a failed treatment for Huntington’s patients,” the official said. “They probably accept or understand on a deep level that their trial failed years ago, and UniQure is making a comparison that is skewed or manipulated in the FDA’s mind, rather than doing the right thing and conducting the correct clinical study.”
The comments mark the latest development in the public dispute between UniQure and the FDA, as the agency has come under fire for a recent series of rejections of drug approval applications, including where some companies accused it of reverting to previous guidance. FDA Commissioner Marty Makary apparently criticized 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 its treatment.
UniQure later accused the FDA of reversing the company’s position that clinical trial data would be sufficient to gain approval. UniQure’s study used an external database, known as an external control, to measure how patients with Huntington’s disease might regress without treatment. UniQure said it would not be possible to conduct a truly randomized, double-blind, placebo-controlled study, considered the gold standard, because it would be unethical to put people through hours-long sham brain surgery.
The FDA official said the agency “never accepted this distorted comparison” and that the FDA “never gave such assurances.” Instead, “the FDA will always say, ‘We need to see the data when we get it.'”
UniQure said in a statement that it was “confident in the strength of the data we submitted to the FDA” and shared “the administration’s goal of developing meaningful treatments for patients suffering from rare diseases.”
“Recent statements made to the press by anonymous FDA sources are highly erratic, unprecedented, and incomplete or completely inaccurate,” the company said. “We do not believe these reflect a fair and faithful reading of the documents we sent or received from the agency.”
The company’s shares rose 18% on Thursday and were down 56% this year as of Thursday’s close.
