$uniQure(QURE)$ uniQure (网页链接#hasComeFromMpArticle=false#source=section%3Amain_content%7Cbutton%3Abody_link%7Cfirst_level_url%3Anews{QURE}) shares fell ~28% on Thursday after FDA Commissioner Marty Makary, in a potential reference to Danish biotech’s gene therapy AMT-130, suggested his agency will not approve drugs that are associated with patient morbidity.
His comments come at a time when uniQure (网页链接#hasComeFromMpArticle=false#source=section%3Amain_content%7Cbutton%3Abody_link%7Cfirst_level_url%3Anews{QURE}) is seeking FDA accelerated approval for AMT-130, which is designed to treat a rare genetically driven neurodegenerative disorder 网页链接#hasComeFromMpArticle=false#source=section%3Amain_content%7Cbutton%3Abody_link%7Cfirst_level_url%3Anews{called Huntington’s disease.}
During an interview with CNBC on rare-disease drug approvals on Thursday, Makary said, "I think there has been a bit of an effort to find a boogeyman to account for certain products that were not approved.”
“For example, there was a product where the researchers drilled a burr hole, literally a hole in people's skulls, to interact inject intrathecally into the ventricle" as part of a randomized controlled trial.
AMT-130 is administered directly into patients’ brain tissue through a neurosurgical procedure. “At the end of the randomization period, it's found no benefit, and yet, this is one of the drugs that we were pressured to approve,” Makary added.
“I have a lot of sympathy for those patients. There's 网页链接{nothing out} there to offer them, but we're not going to go ahead and approve something like that that has morbidity associated with it,” he said.