Travere fails to convince regulators to consider accelerated approval for a rare kidney disease — again
As the old saying goes, ‘If at first you don’t succeed, try, try again.’ The origin of this mantra is disputed but has been widely attributed to Robert the Bruce, the first king of Scotland, as he and his army did battle with invading English forces in the early 14th century.
But what happens if you try a second time and again don’t succeed?
Travere Therapeutics will be asking itself that question after a second attempt to gun for accelerated approval was rejected by the FDA, the company announced Wednesday afternoon. The former Martin Shkreli biotech had attempted to push through its lead drug, sparsentan, to a speedy OK in a rare kidney disease, but regulators prefer the more methodical, full approval route.
The way Travere tells it, interim data from its Phase III DUPLEX study and recent additional eGFR data do not meet the FDA’s “threshold to support an application for accelerated approval in FSGS,” per a press release. Regulators remain open to approving sparsentan based on the DUPLEX trial once it’s complete, the biotech added, recommending the company “pursue traditional approval based on two-year eGFR slope.”
Analysts had been looking for an update here ahead of Travere’s second quarter earnings report, which will take place Thursday morning. Wednesday’s outcome had been forecast under a potential “bear case” scenario by Jefferies’ Maury Raycroft last month.
But investors didn’t appear as concerned as Raycroft, who had predicted a roughly 30% stock drop should this update come through. While Travere shares $TVTX did fall post-market, they only dropped about 10% and remained there before Thursday’s opening bell.
Travere is developing sparsentan to treat focal segmental glomerulosclerosis (FSGS), a rare kidney disease where scar tissue builds up on the kidney’s filters and affects patients’ ability to remove waste from their blood. In February 2021, Travere read out a positive, interim Phase III analysis and pledged to win the first accelerated approval for FSGS.
But that May, US regulators rejected the company’s accelerated approval pitch saying data from the interim readout “would not be adequate” to support a quick OK. At the time, Travere had hoped additional eGFR data could persuade the agency to reconsider, but that did not turn out to be the case.
Instead, Travere will now pursue the traditional approval after the DUPLEX study reads out full data, expected in the first half of next year. If everything turns out positive, it could lead to an NDA filing by the end of 2023, Travere says.
“While we are disappointed that we will not be filing for accelerated approval of sparsentan in FSGS, the eGFR data in the DUPLEX Study have continued to progress in a manner consistent with the profile of sparsentan and we remain confident that the study can support an application for full approval in FSGS next year,” CEO Eric Dube said in a statement.
Elsewhere, the company’s pitch for sparsentan in IgA nephropathy remains on track, Travere added Wednesday, with a PDUFA date on track for Nov. 17. The biotech managed to pursue accelerated approval in this indication, and after a recent FDA meeting, execs say no advisory committee is currently planned.