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TThe future of aducanumab, Biogen’s Alzheimer’s disease treatment, appeared brighter after reviewers at the Food and Drug Administration said they believed the drug was safe and effective enough.
In a lengthy document released Wednesday, FDA staff appear to have endorsed the treatment̵7;s approval, which will become the first new treatment for Alzheimer’s in nearly two decades. The commentary, which provides a first glimpse of how FDA scientists view this controversial treatment, ahead of Friday’s meeting of outside advisers who will make a dropout. The vote is not binding on whether or not to propose 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 are “robust and particularly compelling,” writing FDA critics, confirming the drug’s effect on cognition of Alzheimer’s patients. A second, almost identical study showed no significant benefit, but “on closer examination,” FDA agrees with Biogen’s argument that all data shows aducanumab useful for patients. .
The reviewers did not explicitly appeal for FDA approval, but the tone of their comments seemed to support Biogen’s case.
The FDA review seems to alleviate two major concerns about aducanumab: the fact that its two key studies have different results, and the likelihood that a common side effect informs patients whether they are treated. treatment or placebo.
On the first point, FDA reviewers wrote that an insight into the data revealed that a small number of patients with rapidly progressing Alzheimer’s could have misleading the study. When their results were rejected, the numbers favored aducanumab, which is consistent with other studies on the drug, the critics wrote.
In terms of safety, a frequent side effect of aducanumab is a painful but controllable swelling of the brain called an ARIA. Outside experts were concerned that side effects could undermine the study, informing patients that they had received treatment, and thereby creating research bias. But the FDA’s reviewers said analyzing the data did not show that ARIA biased the test.
However, not all FDA reviewers were convinced by reading Biogen’s data. Buried on page 255 of the agency’s summary, statistical critic Tristan Massie concluded that the only positive test of aducanumab could not outweigh the negative test, writing that “there is no evidence. convincing, substantial about therapeutic effect or slow disease and need another study to validate or deny positive and negative research. “
There are no current drugs available to 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 removing harmful protein clumps called beta amyloid that are thought to be able to destroy the brain. It will be an important medical achievement.
It will also be a big deal for Biogen’s profits. People with mild cognitive impairment or early signs of Alzheimer’s will be most likely to benefit from a drug like aducanumab, if it is put on the market. But even limited usage will translate into blockbuster sales. Cowen estimates that 2.2 million Americans suffer from mild dementia due to Alzheimer’s disease. If a third of these people are treated with aducanumab, estimated at $ 50,000 per year, revenue will reach $ 36 billion per year, according to the health care investment bank.
Abbvie’s Humira was the best-selling drug last year, with sales of $ 19.7 billion. Merck’s Keytruda comes in second, with 2019 revenue of $ 11 billion.
Even if aducanumab is prescribed to a small number of qualified patients, it may still be among the best-selling drugs of all time. Today, Biogen is criticized – and its shares are fined – for selling drugs that are losing market share to cheap generics or to newer, more powerful competitors. Aducanumab, if approved, will boost Biogen’s growth and fundamentally change the stock’s outlook for the better.