 Times article
Alzheimer’s drug, aducanumab, gives first new hope in 20 years
The first new drug for Alzheimer’s disease in nearly 20 years won conditional approval today, offering fresh hope for British patients and millions of others worldwide.
The US Food and Drugs Administration (FDA) said the company Biogen could market the drug, called aducanumab, in the United States, but that it would need to complete a further large clinical trial to confirm its benefit to patients.
Regulators in Europe and the UK are not expected to make any ruling until the autumn but the US decision was hailed as promising by British campaigners.
Professor Bart De Strooper, the director of the UK Dementia Research Institute, said: “With no effective therapies currently available to modify the progression of this devastating condition, this is a major milestone for the millions of people living with Alzheimer’s.” He warned however that “controversy and uncertainty” surrounding trials of the drug might limit the impact of the new treatment.
Biogen’s decision to file for approval was a surprise after clinical trials involving over 3,000 people were stopped early in March 2019. An initial analysis showed the drugs were unlikely to make sufficient improvements to people’s memory and thinking.
However, later that year the company said it had undertaken a new analysis with more data that became available after the trials stopped, focusing on people who had taken the drugs for longer. It said that analysis showed a high dose could slow the decline of cognitive skills and keep people able to carry out daily activities for longer, and requested approval on that basis.
The drug is an antibody designed to target amyloid, a protein that builds up in the brain at an early stage of Alzheimer’s disease.
While treatments work to reduce symptoms for a time, aducanumab aims to slow the underlying disease itself.
The FDA said it was basing its unusual conditional approval on the ability of the drug, also known as Aduhelm, to clear the toxic proteins from the brain. For Biogen to continue marketing the drug, it will need to complete a trial to confirm removing the plaque offers cognitive benefits.
Professor John Gallacher, director of Dementias Platform UK, said: “Although I suspect this was a close decision, the FDA obviously gave aducanumab the benefit of the doubt.
“Apart from the chance for patient benefit, the scientific impact of this decision will be the real-world evidence it creates as the progress of people taking the drug is monitored.” He said it could also act as a catalyst for further drug development, suggesting other companies might “dust down” their own similar drugs for further work.
Many experts are uneasy with the ruling. Dr Caleb Alexander, a drug safety and effectiveness expert at the Johns Hopkins Bloomberg school of public health, who was involved with an initial FDA decision to reject the application in November, told The New York Times that relying on a re-analysis of a trial was “like the Texas sharpshooter fallacy – the idea that the sharpshooter shoots up a barn and then goes and draws a bullseye around the cluster of holes that he likes”.
Speaking before the decision, he warned approval would set a “remarkably dangerous precedent” for not only Alzheimer’s research but also broader regulation of prescription drugs.
The American Geriatrics Society had urged the FDA not to approve the drug, citing concerns including “reliance on a single, incomplete trial as the basis for approval” and the fact that it was not free of side effects.
There are about 850,000 people living with dementia in the UK.
Dr Richard Oakley, head of research at Alzheimer’s Society, said: “It’s promising to see that aducanumab has been approved for use in people with early stage Alzheimer’s disease — the first drug to be approved in nearly 20 years by the US regulatory authorities.
“We await the opinion of the European Medicines Agency and the outcome of any application made to the UK regulatory authorities, to give clarity to people with early Alzheimer’s disease in the UK.” He said that with the drug only likely to benefit a proportion of people in the early stages of the disease, “we must keep searching for drugs for all stages of Alzheimer’s disease and for other types of dementia”, calling on the government to honour a commitment to double dementia research funding. |
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