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unicure A senior official at the U.S. Food and Drug Administration said on a call with reporters Thursday that another study needs to be conducted to prove that the company’s gene therapy “actually helps people with Huntington’s disease.”
The official, who requested anonymity before discussing sensitive information, confirmed that the agency has asked the company to conduct a placebo-controlled trial of the treatment, which is administered directly to the brain. UniQure said this type of research was unethical because it required people to be under general anesthesia for hours, but officials disputed this nature.
“So what’s really going on? UniQure is the latest company to develop a failed treatment for Huntington’s disease patients,” the official said. “They probably recognize or understand on a deep level that their trials failed years ago, and instead of doing the right thing and conducting the right clinical studies, UniQure is making distorted or manipulated comparisons in the FDA’s mind.”
The comments mark the latest development in a messy public battle between UniQure and the FDA, which has seen UniQure come under fire for several recent rejections of drug approval applications, including one that accused the company of reverting to previous guidance. FDA Commissioner Marty McCulley appeared to criticize UniQure’s Huntington disease gene therapy in an interview with CNBC’s Becky Quick last week. McCurry didn’t mention UniQure by name, but he did explain its treatment.
UniQure then accused the FDA of reversing its position that its clinical trial data were sufficient to seek approval. The UniQure study used an external database, known as an external control, to measure how Huntington’s disease patients would decline without treatment. UniQure said it was not practical to conduct a true randomized, double-blind, placebo-controlled study, which is considered the gold standard, because it is unethical to subject people to hours of fake brain surgery.
FDA officials said the agency “never agreed to accept this distorted comparison” and that the agency “never makes any such assurances.” Instead, “FDA will always say, ‘Once we get the data, we’ll have to look at it.'”
UniQure said in a statement that it is “confident in the strength of the data we have submitted to the FDA” and shares “the government’s goal of developing meaningful treatments for patients suffering from rare diseases.”
“Recent statements made to the press by anonymous FDA officials are highly irregular and unprecedented, and are either incomplete or completely false,” the company said in a statement. “We do not believe they reflect a fair and faithful interpretation of the documents we submitted or received from the authorities.”
The company’s shares rose 18% on Thursday, but were down 56% year-to-date as of Thursday’s close.
