- The regulatory authority that recommends whether new drugs should be approved in Europe does not believe Biogen's Aduhelm should be allowed on the market, a position which could further imperil the once-lofty sales expectations for the new Alzheimer's disease treatment.
- In issuing its negative opinion, the European Medicines Agency cited the "conflicting" evidence generated in the main clinical trials that were meant to show Aduhelm can effectively treat patients with early stage Alzheimer's. The agency also took issue with the drug's known side effects — which can include brain swelling and bleeding — and ultimately concluded "the benefits of Aduhelm did not outweigh its risks."
- Breaking into the European market had become more critical for Biogen, given the challenges it's faced trying to sell Aduhelm in the U.S. Though that market now looks out of reach, Biogen said Friday it intends to ask the EMA to re-examine its opinion.
Aduhelm is the first drug ever approved in the U.S. to treat Alzheimer's itself, rather than one of the disease's symptoms.
Analysts had expected that, with an estimated 6 million Alzheimer's patients in the states, such a title would lead to rapid sales. But that hasn't been the case so far. Biogen recorded just $300,000 in Aduhelm sales between July and September, well below the $16 million that Wall Street forecasted.
The slow uptake has several causes. Many experts, including the FDA's own advisers, don't view the mixed clinical trial data that Biogen generated as proof its drug actually works. Some doctors are even holding off on prescribing Aduhelm until they get a more in-depth look at the clinical trial data that ultimately led to its approval. Biogen said it plans to publish such data in a peer-reviewed journal, but that hasn't happened yet.
Insurance coverage has also been a hangup, as Aduhelm's average list price of $56,000 a year is far above what many analysts and payers envisioned.
With a few exceptions, insurance providers appear to be waiting on the Centers for Medicare and Medicaid Services to set the tone. The agency — which, through Medicare, covers the vast majority of patients likely to receive Alzheimer's treatments — is currently assembling a single national policy for drugs that work like Aduhelm. A draft of that policy should arrive in January, with a final version expected by April.
But until payers iron out policies, it will be difficult for most doctors to prescribe Aduhelm. As of Oct. 20, Biogen estimated that around 120 treatment sites were infusing Aduhelm, representing a small portion of the 900 or so the company had been targeting since the drug's launch in June.
"We are obviously disappointed with the delayed uptake of Aduhelm in the U.S.," Biogen CEO Michel Vounatsos said during the company's third quarter earnings call in October.
Amid the challenges in the U.S., Biogen had hoped an approval in Europe would give its drug a much-needed boost. Those hopes dimmed last month, however, when the EMA's Committee for Medicinal Products for Human Use signaled that it's view on Aduhelm wasn't positive.
The EMA is now officially recommending the European Union's executive branch reject the Aduhelm marketing application. On its end, Biogen plans to request a re-examination of the negative opinion. Once the request is received, the EMA will have about two months to address it.
"European patients deserve access to innovative treatments for Alzheimer's disease," Priya Singhal, Biogen's head of global safety and regulatory services, and interim head of research and development, said in a Dec. 17 statement.
Biogen shares were up about 1% Friday morning, to trade close to $238 apiece. Since Aduhelm's approval in early June, though, shares of the Cambridge, Massachusetts-based biotech have tumbled roughly 40%.