Amicus CEO John Crowley’s latest pitch to put a new drug on a pathway to accelerated approval has run into a hurdle at the FDA. Crowley acknowledged in a statement out early Monday that a meeting with regulators failed to generate much enthusiasm for a quick review of AT-GAA (ATB200/AT2221) based on what they’ve seen so far in a Phase I/II trial.
But Crowley $FOLD, who recently overcame the odds in winning a reversal of the FDA’s decision to initially reject Galafold, isn’t about to stop now. The company says it plans to keep pressing, just as it did earlier this summer after the EMA also held up a stop sign to a conditional approval.
Amicus Therapeutics intends to continue to generate data to support further discussions on a potential pathway for accelerated cpproval with the FDA in 2019, including:
- Data from up to 10 additional ERT-switch patients in a new Cohort 4 as part of the ongoing Phase 1/2 study (data expected in 2019)
- Presentation of longer-term clinical data out to 18-months for the 19 original Phase 1/2 patients (data expected in 2H 2018)
- Completion of a retrospective natural history study in approximately 100 ERT-treated Pompe patients (data expected in 2H 2018).
That’s the same game plan Amicus outlined when the EMA earlier closed the door on their quest to gain a conditional approval in Europe.
There have been waning expectations that the FDA would come through here on little more than biomarker data. But with the new Trump administration — represented by FDA commissioner Scott Gottlieb — celebrating fast new OKs while hammering any prospective price increases, hope lingered. And Crowley isn’t known for taking no as an answer.
Leerink’s Joseph Schwartz, who led the sell side cheerleaders, has been keeping the flame burning. He noted recently:
Galafold approval could reflect a more flexible FDA that is willing to accelerate the regulatory process in the US.
Not this time. Or at least, not yet. And Schwartz this morning says the snub will likely lead to a lengthy delay for this drug ahead of any approval.
We had previously assigned 50% probability of a 2019 mkt. entry but we are now pushing out the commercial launch to 2022E, with a 75% PoS.
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