POGO Implicates Gottlieb in Amicus Drug’s FDA Review Advance
The Project On Government Oversight (POGO) is questioning how biotech executive John F. Crowley, who president Trump profiled as an example of speeding drug development in a February address to Congress, was able to advance a stalled experimental drug called migalastat to a fast track review at FDA. Crowley’s success included a personal appeal to then-newly confirmed FDA commissioner Scott Gottlieb, according to documents obtained by POGO.
Before becoming the agency head this year, Gottlieb was a venture partner at New Enterprise Associates, a venture capital firm with investments in Amicus Therapeutics, where Crowley is the CEO. “Crowley’s outreach to Gottlieb offers a glimpse of the drug industry’s interactions with the FDA,” a POGO blog post says. “Concerns about the agency’s relationship with companies it oversees are nothing new, but, given his industry background, Gottlieb’s appointment reinforced them.
In May, Crowley sent Gottlieb a handwritten note that mentioned an earlier conversation between the two: “As discussed, the hopes and well being of so many living with rare, devastating diseases rests now with your great leadership and wisdom. Thanks for your attention to the attached. All the Best, John.”
According to POGO-obtained documents, Crowley attached a formal letter that explained how Amicus hoped the agency would review the experimental drug based on data already in hand instead of requiring another study. Crowley wrote that conducting the additional study was “not feasible in a reasonable amount of time.” It would take another five to seven years, he said.
Two months later, in July, Amicus announced that FDA had abandoned a requirement imposed on the company to conduct an additional Phase 3 study to assess gastrointestinal symptoms with migalastat for Fabry disease, a move that opened a path for an NDA filing (see story). Amicus said it would base its NDA, which was filed 12/14, on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies. “Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke,” it said. Migalastat works by “stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations,” it explained. “An amenable mutation is one that is responsive to therapy with migalastat based on a proprietary in vitro assay.”
POGO notes that the documents it obtained through the Freedom of Information Act do not contain any instructions from Gottlieb to FDA staff on migalastat, and do not signal that Gottlieb played any role in reversing the clinical trial requirement
Asked to comment by EndPoint News, an FDA spokeswoman said Gottlieb was removed from the matter and he was not aware of the letter. “Product-specific correspondence that is sent to Dr. Gottlieb by external parties is referred to the relevant centers as a matter of routine procedure. Further, decisions on whether to accept to file an application or grant a drug application priority review (or any of the other pathways for drug review) is based on specific criteria applied to the product’s data and determined by appropriate FDA career staff.”