An analyst report stating that Roche will seek early FDA approval for its Alzheimer’s disease candidate gantenerumab appears to have jumped the gun, according to comments from the Swiss pharma giant.
Jefferies stated in a research note that Roche had met with FDA regulators last week and would be following in Eli Lilly's footsteps to file the candidate under an accelerated review pathway.
But Roche told Fierce Biotech they would not "comment on speculations." Instead, the company is continuing with a phase 3 clinical trial for gantenerumab and more details will be shared when they are available. Discussions with the FDA and other regulatory authorities are being done "when appropriate."
The news was the latest in a saga that began when the FDA approved Biogen's Aduhelm for Alzheimer's disease under the accelerated approval lane. The therapy is the first approved for the devastating neurological disease in decades and has set off a firestorm of speculation over which companies might leap through the door cracked open by Biogen.
Aduhelm was approved by the FDA on the strength of data for a surrogate endpoint, thereby raising the possibility that developers of other Alzheimer’s candidates could get to market before completing big pivotal studies that show clinical benefit.
Lilly responded by committing to seek accelerated approval for donanemab later this year; however, Mizuho analysts said in a June 24 note that the company and the FDA "are not yet fully aligned" on the plan to submit the therapy. Donanemab was granted a breakthrough therapy tag, kicking off excitement that the drug may follow in Aduhelm's footsteps.
Given the precedent Aduhelm set, Mizuho still thinks the FDA will accept and ultimately approve Lilly's drug.
As for gantenerumab, Roche brought the drug candidate back from the dead in 2018, four years after it failed a phase 3 study, in the belief that a much higher dose may be more effective.
The 2014 flop, coupled with the later failure of a Washington University School of Medicine-sponsored study, leaves sizable doubts about whether gantenerumab can improve clinical outcomes. However, Roche has linked the new phase 3 dose to the removal of amyloid-beta plaque. In a post-Aduhelm world, data on amyloid-beta plaque removal could be enough to win accelerated approval.
Jefferies said that if gantenerumab is filed in the near future and granted priority review and a fast track designation, the drug could be headed for shelves under an accelerated approval late in the first quarter of 2022. This would be a year earlier than expected.
Roche is expecting data from the late stage trial to arrive in the second half of next year, putting the Jefferies timeline firmly out of reach if the Swiss pharma intends to wait.
If gantenerumab comes to market, it could have advantages over the potential pool of rivals such as Biogen’s Aduhelm. Notably, gantenerumab is administered subcutaneously over five minutes, making it quicker and easier to deliver than intravenous therapies such as Aduhelm. The Jefferies analysts forecast gantenerumab sales of $3 billion but assign a 0% probability of success to the candidate.
Editor's note: An earlier version of this story reported that Roche will be filing for accelerated approval for gantenerumab imminently. The story has been updated to include commentary from Roche.