Updates can be found at the end of the article; the most recent update is from April 22, 2022.
Two House committees sent a 13-page letter to acting US Food and Drug Administration Commissioner Janet Woodcock on Wednesday (September 1) as part of an investigation into the recent accelerated approval of a controversial drug for Alzheimer’s disease.
The letter—signed by representatives Carolyn B. Maloney (D-NY), chairwoman of the Oversight and Reform Committee, and Frank Pallone Jr. (D-NJ), chairman of the Energy and Commerce Committee—requested information about who was involved in the approval of the Biogen-produced drug and how the decision was made.
The drug—called Aduhelm—received accelerated approval from the agency on June 7, making it the first FDA-approved drug for Alzheimer’s in nearly 20 years. Aduhelm is supposed to remove amyloid-β plaques that accumulate in the brain, although some experts say plaque removal doesn’t slow disease progression and may carry the risk of causing bleeding or swelling in the brain, according to The New York Times.
Two late-stage clinical trials on Aduhelm were stopped in 2019 due to a lack of clear evidence of the drug’s efficacy, although Biogen, the company behind the drug, later said a reanalysis of the data showed it had benefits. A panel of outside experts recommended against approving the drug, and according to the Times, a former senior medical director at Biogen also opposed the approval.
“We are concerned by apparent anomalies in FDA’s processes surrounding its review of Aduhelm,” says the letter, according to STAT. “We are also concerned by reports of unusual coordination between FDA and Biogen throughout the drug’s approval process.” The same committees had requested documents from Biogen in July pertaining to its assessments of Aduhelm and the regulatory process.
According to the Times, the letter requests information about undocumented meetings between FDA officials and Biogen representatives, and asks whether agency officials collaborated with Biogen on projects or proposals while the drug was awaiting approval.
Despite the approval, the Times reports that several major medical centers in the country are refusing to offer Aduhelm, including Providence, Mount Sinai Health System, and the Cleveland Clinic. The treatment costs about $56,000 a year and requires monthly intravenous infusions.
In response to the letter, Biogen said in a statement on Thursday, “We are continuing to cooperate with the committees’ investigation and to produce documents in response to the committees’ requests,” according to the Times.
Update (November 17, 2021): An advisory panel of the European Medicines Agency voted against the approval of Aduhelm, according to an announcement from Biogen posted today. The EMA, which will meet next month to discuss the new Alzheimer’s drug, is likely to follow that recommendation, Reuters reports. Meanwhile, Biogen Head of Research & Development Alfred Sandrock, who spearheaded the development of Aduhelm, will retire by the end of the year, the company said Monday (November 15).
Update (December 17, 2021): The European Medicines Agency (EMA) announced today that it is recommending against authorizing Aduhelm, noting among other reasons that “although Aduhelm reduces amyloid beta in the brain, the link between this effect and clinical improvement had not been established.” In response, Biogen announced that it will seek a re-examination of the opinion by the EMA’s Committee for Medicinal Products for Human Use.
Update (December 22, 2021): A panel of Japan’s Ministry of Health, Labor and Welfare has declined to approve Aduhelm for use in that country, concluding that available data do not demonstrate that the drug slows cognitive decline in patients with Alzheimer’s, Kyodo News reports.
Update (April 8, 2022): The Centers for Medicare and Medicaid Services has finalized a proposal that coverage for Aduhelm will be limited to patients participating in clinical trials, multiple outlets report. Agency representatives say the policy aims to protect patients from potential side effects while enabling further evidence to be collected about the treatment’s effectiveness.
Update (April 22, 2022): Biogen has withdrawn its application for marketing authorization of aducanumab from the European Medicines Agency, the company announced today.