CHMP minded to turn down Alzheimer's drug Aduhelm, says Biogen

Biogen and Eisai have said that the EMA's human medicines committee looks unlikely recommended approval of their Alzheimer's drug Aduhelm when it comes up for discussion next month.

The CHMP had a "negative trend vote" on the drug at a meeting last week which included an "oral explanation" of Aduhelm (aducanumab), which was approved in controversial circumstances by the US FDA earlier this year.

Shares in Biogen started to slide in pre-market trading after the update was issued, just two days after the biotech announced the sudden departure of long-serving R&D head Al Sandrock.

While Sandrock isn't due to step down until the end of the year, it was left to interim successor Priya Singhal – Biogen's head of global safety and regulatory sciences – to comment on the development.

"While we are disappointed with the trend vote, we strongly believe in the strength of our data and that aducanumab has the potential to make a positive and meaningful difference for people and families affected by Alzheimer's disease," she said.

The preliminary vote does not mean that the CHMP finalised its thinking on Aduhelm's marketing application in the EU, and the EMA doesn't have to follow its panel's advice even if it decides to reject the drug.

That said, given the ongoing debate about Aduhelm's efficacy, and the high price charged by its developers for the drug in the US, there seems little chance of a positive outcome for Biogen and Eisai at the December CHMP meeting.

Biogen said it would "continue to engage with the EMA and CHMP as it considers next steps towards the goal of providing access to aducanumab to patients in Europe."

Several hospital systems in the US have already decided not to provide the drug, while some payers are refusing to cover its cost given doubts about not only its efficacy but also its safety, mainly centring on the risk of a form of brain swelling known as ARIA that has been seen in some patients.

So far, only a few patients are getting aducanumab, with Biogen reporting sales of $1.6 million in the second quarter of the year, falling to just $300,000 in the third against analyst expectations of $12 million.

Biogen has said it expects sales to be "minimal" through the remainder of the year and potentially into 2022 as it waits for the outcome of a review by the Center for Medicare and Medicaid Services (CMS), which isn't due to deliver recommendations on reimbursement until next year.

Now, it looks like it won't be able to rely on European take-up to get the Aduhelm programme back on track. The drug has also been filed for approval in Japan.