In a lengthy document released Wednesday, FDA staff appeared to endorse approval of the treatment, which would become the first new treatment for Alzheimer’s disease in nearly two decades. The commentary, which provides the first glimpse into how FDA scientists view the often-debated treatment, comes ahead of a Friday meeting of outside advisers, who will cast a non-binding vote on whether to recommend the treatment. approval of aducanumab.
Biogen shares rose nearly 45% to $ 355, adding $ 17 billion to the company’s market value.
Biogen’s data from a large clinical trial was “robust and exceptionally convincing,” wrote the FDA reviewers, endorsing the drug’s effects on cognition in Alzheimer’s patients. A second, almost identical study did not show any significant benefit, but “upon further examination” the FDA agreed with Biogen’s argument that the sum of all data suggests aducanumab is useful for patients.
publicityCritics did not explicitly call for FDA approval, but the tenor of their remarks seemed to support Biogen’s case.
The FDA review appeared to alleviate two major concerns about aducanumab: the fact that its two pivotal studies had different results and the possibility that a common side effect would inform patients whether they were receiving treatment or a placebo. .
On the first point, FDA critics wrote that an extensive review of the data suggested that a small number of patients with rapidly progressing Alzheimer’s disease may have skewed the study. When their results are suppressed, the numbers favor aducanumab, which is consistent with other studies on the drug, the reviewers wrote.
On the safety side, a common side effect of aducanumab is a painful but manageable swelling of the brain called ARIA. Outside experts were concerned that the side effect could effectively override the blindness of the study, informing patients that they had received the treatment and thus creating a bias in the study. But FDA critics said an analysis of the data did not suggest ARIA biased the trial.
However, not all FDA reviewers were convinced by Biogen’s reading of the data. Buried on page 255 of the agency’s briefing, statistical critic Tristan Massie concluded that the single positive aducanumab trial cannot outperform the negative, writing that “there is no convincing and substantial evidence for it. effect of treatment or of slowing the disease and that another study is needed to confirm or deny the positive and the negative study. ”
There is currently no drug that can slow the cognitive and functional decline that occurs in people with Alzheimer’s disease. If approved, aducanumab would be the first drug to reduce cognitive decline by targeting and eliminating clumps of a toxic protein called beta-amyloid that is believed to destroy the brain. It would be a significant medical achievement.
It would also be a big problem for Biogen’s bottom line. People with mild cognitive impairment or early signs of Alzheimer’s disease would be the most likely to benefit from a drug like aducanumab, if it hits the market. But even limited use would result in successful sales. Cowen estimates that 2.2 million Americans suffer from mild dementia due to Alzheimer’s disease. If a third of these people were treated with aducanumab, valued at $ 50,000 per year, sales would reach $ 36 billion per year, according to the health investment bank.
AbbVie’s Humira was the top-selling drug last year, with sales of $ 19.7 billion. Merck’s Keytruda finished second, with 2019 revenue of $ 11 billion.
Even if aducanumab is prescribed to a fraction of eligible patients, it could still be among the best-selling drugs of all time. Today, Biogen is being criticized – and its stock penalized – for selling drugs that are losing market share to low-cost generics or newer, more powerful competitors. Aducanumab, if approved, would stimulate the growth of Biogen and fundamentally change the outlook for action for the better.