Drug Development, FDA

FDA orders a rare retreat on Amicus’ migalastat, dropping PhIII demand and signaling a major shift for drug developers

Megan Crowley is recognized by President Donald Trump during his first address to a joint session of Congress on February 28, 2017. At 15 months old, Crowley was diagnosed with Pompe Disease and not expected to live more than a few short years. AP Images

Amicus Therapeutics $FOLD announced early Tuesday that the FDA has dropped its demand for a new Phase III study of its Fabry disease drug migalastat after it was stiff-armed by unsatisfied regulators last fall — then under a different administration with a different FDA commissioner.

Instead of pursuing a new and expensive late-stage study, Amicus CEO John Crowley tells me the company has the green light to file again with the data at hand. And based on discussions with the FDA, he adds, the company is shooting to file in the 4th quarter, with an accelerated approval that could arrive in 2018, potentially slicing years off the process.

“These are the days it’s good to be a biotech CEO,” says Crowley, who has been on a roller coaster ride at the FDA over the past year after being forced to walk back his own plans last November.

John Crowley

There is no guarantee of an approval here, but the agency’s sudden volte-face clearly signals a new tone that vastly improves the biotech’s shot at an OK — at least in so far as we hear it from Crowley. And that could have a major influence on other developers tackling the same task.

Investors cheered on the sudden change in fortunes at Amicus, bidding up shares by 43% in pre-market trading Tuesday.

Crowley has never made a secret of the fact that he felt the drug — approved in Europe last year — was ready to go in the US after it completed the pivotal program. The FDA, though, rejected his application, saying regulators wanted more safety data than the Europeans needed.

That rejection became a cause celebre of sorts in DC, as President Donald Trump turned to Crowley’s daughter, Megan, who suffers from Pompe disease, and called out the FDA for the “slow and burdensome” process involved in new drug approvals during an address to Congress.

“Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” Trump said at the time. “If we slash the restraints, not just at the FDA, but across our government, then we will be blessed with far more miracles like Megan.”

Crowley picked up on that change in thinking at the White House in a blog post at the time in which he said: “The FDA’s regulation of the orphan development process is becoming less flexible, less efficient and less patient-centered.”

I asked Crowley what had changed between the setback last fall and the new, soft green light that the FDA has switched to.

First and foremost, Crowley hit the theme that the company had made a convincing case with what was available. That includes new data and analysis that was made possible after the rejection. The company started from the beginning to marshal the evidence needed to meet the agency’s gold standard for an OK. That, he says, was absolutely key, ultimately winning the day.

In addition, he says there’s also a new framework for drug reviews that’s been gathering steam at the agency.

“I do think, candidly, that there is an emerging framework in rare diseases,” he says, pointing to new approvals for Biomarin on Batten disease as well as a novel label extension for Vertex based on their in vitro assay.

Scott Gottlieb appears before a US Senate subcommittee, June 20, 2017 AP Images

You can bet, though, that there will be plenty of sentiment suggesting that a change of administrations and a new FDA commissioner in Scott Gottlieb made a telling difference for an abrupt about-face that is extraordinarily rare to see at the FDA.

Whatever the cause, though, instead of preparing to gather new data that wouldn’t be available until 2019, Crowley is back to making launch plans in the US. That will probably require a commercial group of about 50, he says, compared to the 80 needed to address a more complex and challenging payer environment in Europe.

Biotech has largely cheered on new FDA commissioner Scott Gottlieb after he won confirmation by the Senate. He promised to turn to new technologies to help accelerate new approvals, but vowed to stick with the industry gold standard that has governed regulators thoughts on safety and efficacy date.

So did the FDA just soften its standards or wake up to the fact that the drug should never have been rejected in the first place?

Let’s start the discussion.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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