\n In a highly unusual move for a normally buttoned-up regulatory agency, a senior official with the US Food and Drug Administration held a call with reporters Thursday to defend the agency\u2019s recent decision on an experimental medicine, calling it a \u201cfailed therapy\u201d and railing against outside critics.\n <\/p>\n
\n The call focused on the FDA\u2019s decision<\/a> to ask for an additional clinical trial from the biotechnology company UniQure before greenlighting it to apply for approval of its gene therapy for Huntington\u2019s disease, a fatal neurodegenerative disorder. Patients and the company had hoped<\/a> the therapy could move into an FDA review based on existing evidence, a hope that was dashed<\/a> Monday.\n <\/p>\n \n But the regulator, who spoke on the condition of anonymity, also attacked outside scientists who serve on advisory committees for the agency, saying they \u201cdo not do their homework\u201d and may be motivated by financial conflicts of interest. The official additionally singled out Dr. Janet Woodcock, a retired FDA veteran <\/strong>who called the UniQure decision \u201ctruly evil\u201d in an interview<\/a> with the New York Times this week.\n <\/p>\n \n \u201cDr. Woodcock is an esteemed regulator,\u201d the official said, \u201cand as such, there are many commenters who I don\u2019t expect better from, but Dr. Woodcock, I do expect better from.\u201d\n <\/p>\n \n Woodcock declined to comment Thursday on the FDA official\u2019s comments, saying she believed that the FDA should review the application and give it a public hearing.\n <\/p>\n \n The FDA call represented a tense crescendo in a series of moves that have raised questions about whether the regulator is changing goalposts when it comes to drug approvals.\n <\/p>\n \n Numerous companies have claimed that recent actions by the regulator ran counter to prior guidance, slowing approvals of new medicines even as the agency has been touting<\/a> ways it plans to speed up the process.\n <\/p>\n \n Last month, the agency faced a firestorm of criticism for initially refusing<\/a> to review an application from Moderna for an mRNA flu vaccine before ultimately accepting the application with some adjustments. It held a similar media call then with a different senior official to defend itself.\n <\/p>\n \n The FDA has also been criticized for recent moves on rare disease drugs from Atara Biotherapeutics and Pierre Fabre Pharmaceuticals<\/a>, Regenxbio<\/a> and others.\n <\/p>\n \n On Thursday, the FDA official disputed news coverage<\/a> and statements from UniQure suggesting that the agency is requiring the company run a clinical trial involving a fake 10-hour brain surgery as a placebo control for the gene therapy<\/a>, which is given as a one-time infusion administered directly into the brain in an eight- to 10-hour procedure.\n <\/p>\n \n \u201cDoes the sham intervention require 10 hours of anesthesia? No, it does not,\u201d the official told reporters. \u201cIt takes under 30 minutes of anesthesia. \u2026 Did FDA ask UniQure to drill holes in skulls? No, they just need to make one to three nicks in the scalp.\u201d\n <\/p>\n \n The official said the agency requires placebo-controlled trials to ensure that the product is not \u201csnake oil\u201d and argued that it\u2019s longstanding FDA policy to require them for Huntington\u2019s disease treatments.\n <\/p>\n \n Woodcock told CNN that she believes the FDA should review UniQure\u2019s therapy.\n <\/p>\n \n \u201cFor such a terrible disease and people who volunteered to have this therapy put into their brains, they owe it to the community to review and have a public discussion,\u201d Woodcock said. \u201cThey promised \u2018radical transparency.\u2019\u201d\n <\/p>\n \n She noted that she doesn\u2019t have an opinion on the company\u2019s data itself, as she hasn\u2019t seen it. \u201cThis is more about the process.\u201d\n <\/p>\n \n UniQure had hoped to make a submission to the FDA using data from an earlier trial<\/a> that suggested its therapy slowed disease progression compared with an external control group made up of people with Huntington\u2019s who weren\u2019t in the study.\n <\/p>\n