A European Medicines Agency panel voted against Biogen's new Alzheimer's drug Aduhelm at a recent meeting, indicating the controversial treatment could be rejected by the regulator next month.
Biogen acknowledged the negative vote in a Wednesday statement and said it would continue to work with the EMA "as it considers next steps towards the goal of providing access" to Aduhelm.
The panel, known as the Committee for Medicinal Products for Human Use, is expected to conclude its review of the drug at a meeting set for Dec. 13 to Dec. 16. Unless the committee reverses its position, Aduhelm is unlikely to secure EMA approval.
"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," said Priya Singhal, head of global safety and regulatory sciences at Biogen and, as of this week, the company's interim head of R&D. On Tuesday, Biogen unexpectedly announced that its current R&D head and top scientist, Al Sandrock, who championed Aduhelm throughout its development, would retire at the end of the year.
The "negative trend vote" from the CHMP — a sort of straw poll ahead of an official decision — is another blow to Biogen, which has seen Aduhelm become mired in debate over its effectiveness and the way in which it was approved in the U.S.
A federal investigation is now underway into how the Food and Drug Administration applied its standards for so-called accelerated approval to Aduhelm and, since the drug's June 7 approval, several insurers and high-profile treatment centers have refused to cover or administer the drug.
Between July and September, Aduhelm's first full quarter on the U.S. market, sales of the drug totaled just $300,000, far short of the $16 million analysts on Wall Street had expected. Biogen does not expect sales to pick up until Medicare decides whether to cover Aduhelm broadly, a verdict expected by early next year.
Should the EMA follow through on the vote and reject Aduhelm, expectations for Aduhelm sales would shrink further, although analysts view the U.S. as the drug's principal market.
"While somewhat disappointing at face, we still view the U.S. opportunity as representing the bulk of Aduhelm's value, and it was hard to see the EMA going out on a limb and approving [the drug] on its current dataset," wrote Paul Matteis, an analyst at Stifel, in a Nov. 17 note to clients./p>
A positive EMA review, which precedes a marketing authorization from the European Commission, is rarely the final word for a drug launch in the EU. Drugmakers must also negotiate with member country health systems over reimbursement, which is typically lower than in the U.S.
Part of that equation in many cases is a cost-effectiveness evaluation, which weighs a drug's costs against its benefits and potential harms. In the U.S., Biogen charges $56,000 a year for Aduhelm, a much-criticized price that's far higher than what the Institute for Clinical and Economic Review, an independent watchdog group, calculated would be cost effective.
Aduhelm is the first new Alzheimer's drug approved by the FDA in nearly two decades, and the only medicine that's meant to treat what many believe to be the disease's underlying cause. But its clearance was particularly contentious, as results from the two large clinical trials testing Aduhelm produced conflicting results. One trial showed treatment modestly slowed the cognitive decline that's characteristic of Alzheimer's, while the other found no difference between Aduhelm and a placebo.
Moreover, the results of both trials were the product of a reassessment that took place after Biogen originally announced in 2019 that each had failed, making the company's conclusions more tenuous. The FDA, however, worked unusually closely with Biogen to evaluate the data, a collaboration that stemmed in part from an off-the-books meeting between Sandrock and a top agency official that was reported by STAT News.