How did biotech executive John F. Crowley, who President Donald Trump singled out for praise in a February address to Congress, move a stalled experimental drug called migalastat onto a fast track for review at the Food and Drug Administration this year?
Crowley’s efforts to advance the drug included a personal appeal to Trump’s newly confirmed FDA commissioner, Scott Gottlieb, who is himself a veteran of the biotech industry, documents obtained by the Project On Government Oversight show.
Before becoming FDA Commissioner, Gottlieb was a venture partner at New Enterprise Associates. That venture capital firm was a major investor in the company Crowley heads, Amicus Therapeutics, according to securities filings.
Crowley’s outreach to Gottlieb offers a glimpse of the drug industry’s interactions with the FDA. Concerns about the agency’s relationship with companies it oversees are nothing new, but, given his industry background, Gottlieb’s appointment reinforced them.
On May 16, 2017, Crowley sent Gottlieb a handwritten note addressing the commissioner on a first-name basis.
“Scott, Congratulations, again!” Crowley began.
Alluding to an earlier conversation between the two, the note flattered Gottlieb.
“As discussed, the hopes and well being of so many living with rare, devastating diseases rests now with your great leadership and wisdom.”
Then the note got down to business.
“Thanks for your attention to the attached. All the Best, John”
“The attached” was a formal “Dear Dr. Gottlieb” letter bearing the same date and explaining how Amicus hoped the FDA would consider the experimental drug based on data already in hand instead of requiring another study. Crowley wrote that conducting the additional research was “not feasible in a reasonable amount of time.” It would take another five to seven years, he said.
Crowley closed by thanking Gottlieb for his “extraordinary sacrifice in serving in this important role.”
POGO obtained the records from the FDA through the Freedom of Information Act.
Another document provided by the FDA contains internal tracking information indicating that, on May 18, material Crowley sent Gottlieb about migalastat was referred to a staff member in the FDA unit that had called for the additional study.
Weeks later, on July 11, Amicus announced a major breakthrough in its quest for FDA approval.
“An additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission,” the company news release said.
In other words, the FDA had given Amicus a green light to submit a so-called new drug application asking the FDA to approve the product for sale in the United States.
"This guidance from the FDA marks a tremendous step forward,” Crowley said in the company news release. “Today is a seminal moment in the development of migalastat,” he added.
The company’s stock price rose by 25.9 percent that day.
The announcement marked a triumphant reversal for Amicus. Months earlier, on November 28, 2016, Amicus announced that it was resigned to collecting additional data for the FDA.
"While we believe that the totality of the data from our studies with migalastat support the submission of a new drug application today, we acknowledge the FDA's position that accelerated approval . . . is not currently an option,” Crowley said in the 2016 news release.
Migalastat is meant to treat Fabry disease, a genetic disorder that can lead to heart attacks, strokes, and kidney damage, according to medical references.
Crowley is a biotech entrepreneur whose family story has been the subject of the movie Extraordinary Measures and the book The Cure: How a Father Raised $100 million and Bucked the Medical Establishment in a Quest to Save His Children.
Crowley helped develop a drug that saved his daughter Megan from a rare and fatal condition called Pompe disease.
John and Megan Crowley were guests of the President when Trump addressed Congress in February and argued for slashing regulatory restraints. Trump cited Megan Crowley as a medical miracle and said the FDA’s “slow and burdensome approval process” was standing in the way of similar advances. (As POGO reported, the FDA approved Myozyme, the drug for Pompe disease, despite FDA staff members’ concerns that “significant safety information” about that drug had been “obscured.”)
Weeks after that address, Crowley authored an article in the Observer praising Trump’s choice of Gottlieb to head the FDA. “Dr. Gottlieb’s nomination is a great day for science, for medical innovation and above all, for patients and families living with life threatening and disabling conditions,” Crowley wrote.
As POGO reported in April, Gottlieb’s financial disclosure statement listed line after line of payments from drug and biotech companies. He served on company boards and in executive roles, worked as a consultant to pharmaceutical companies, and collected speaking fees from firms such as Merck, Biogen, and Johnson & Johnson.
The documents POGO obtained through the Freedom of Information Act do not contain any instructions from Gottlieb to FDA staff on migalastat. Nor do they contain any reply from Gottlieb to Crowley.
Neither Gottlieb nor Crowley responded to inquiries from POGO. A spokeswoman for New Enterprise Associates did not respond to messages, either.
On September 19, Amicus announced another breakthrough at the FDA. The FDA granted migalastat a special status that is meant to expedite the agency’s review. The designation is known as “Fast Track."