Frankfurt The US Food and Drug Administration (FDA) has granted full approval for the first time for a therapy that slows the progression of Alzheimer’s disease. This was announced by the authority on Thursday evening. The drug Leqembi, developed by the US biotech company Biogen and the Japanese pharmaceutical company Eisai, is considered a milestone in the treatment of the deadly disease.
Previously available drugs could only treat symptoms, such as restlessness, but did not change the course of the disease, which leads to memory loss and eventual death. In a large clinical study with almost 1800 participants, Eisai and Biogen were able to show that Leqembi can reduce cognitive decline in the treated patients by 27 percent compared to the comparison group.
The need for effective treatment is great: According to the World Health Organization, there are around 55 million people with dementia worldwide, around two thirds of whom suffer from Alzheimer’s disease. The number of sick people in Germany is estimated at more than 1.5 million people, in the USA it is said to be more than six million.
A market worth billions should open up for an effective drug. Full approval in the USA could now be the key. The FDA gave the provisional green light back in January. But the state health systems have promised that they will cover the costs of the treatment after full approval. Leqembi will eventually be offered there at a price of $26,500 per year.
“We have applied for approvals in many countries and are working on making Leqembi widely available as a new treatment for Alzheimer’s,” said Biogen boss Christopher Viehbacher in an interview with the Handelsblatt. If the process goes according to plan, the drug could be approved in Europe in the first quarter of next year. “Leqembi could then be launched in the first European countries by the middle of the year,” says Viehbacher.
“Leqembi could then be launched in the first European countries by the middle of the year”
(Photo: Reuters)
In Alzheimer’s disease, nerve cells die. Protein deposits, so-called amyloid plaques and tau fibrils, form in the brain of those affected. The processes are not yet fully understood.
Research in this area has so far been characterized primarily by setbacks. Hundreds of drug candidates developed over the past decades have failed. More and more large pharmaceutical companies had withdrawn from this therapeutic area because of the high risk.
The French company Sanofi also decided against entering into Alzheimer’s research more than a decade ago. Boss back then: Christopher Viehbacher. “You didn’t know what could really work,” says the Franco-German manager today.
Alzheimer’s: Many pharmaceutical companies failed in research
However, Biogen entered into this “high-risk bet”, as Viehbacher calls it, together with its partner Eisai. Both companies have invested billions in research and development. Their product Leqembi, which contains the active ingredient lecanemab, is an antibody that targets the amyloid beta plaques in the brain.
Many companies have also failed with this research approach. Most recently, it was the Swiss pharmaceutical company Roche that had to announce last November that its hopeful ganterumab had failed to prove its effectiveness. And the antibody aducanumab, also developed by Biogen and Eisai, which was provisionally approved by the FDA in mid-2021 against the advice of a panel of experts, had also not provided statistically clear results in clinical studies.
At Leqembi, however, the experts unanimously approved FDA approval. The study provided clear evidence of clinical benefit, said panel member Merit Cudkowicz, a neurologist at Harvard Medical School.
From a researcher’s point of view, the data from the Leqembi study have for the first time confirmed that the amyloid plaques are a real target for the treatment of Alzheimer’s – and that removing them with medication can counteract the damage to brain cells. A thesis that was very controversial in the meantime in the professional world because of the many setbacks.
Lecanemab is only effective in the early stages of Alzheimer’s
Christian Haass, head of the department for neurodegenerative diseases at the LMU Munich, had already spoken of a “turning point” in the study results presented in autumn. In the meantime, the US company Eli Lilly has also achieved positive study data with its amyloid antibody donanemab, which is considered further confirmation of the amyloid hypothesis.
Nevertheless, despite the research success, there are also limitations. “Even this active ingredient cannot stop the decline in mental performance caused by Alzheimer’s disease, but it can delay it a little,” says Alexander Kurz, board member of the German Alzheimer Society.
The Alzheimer’s drug costs around $26,500 a year in the United States.
(Photo: via REUTERS)
In addition, lecanemab is only suitable for patients in an early stage of the disease who only have very slight limitations in their brain performance. In most of those affected in Germany, however, the diagnosis is only made at a much later point in time. There are also side effects such as brain swelling or microbleeding. Patients must therefore be accompanied by imaging diagnostics in the first few weeks of treatment.
The Italian neurologist Carlo Colosimo from the University Hospital of Terni recently even expressed doubts to the Reuters news agency as to whether lecanemab is really clinically meaningful for patients.
Although the study results are statistically significant, it would not be surprising if the European Medicines Agency (EMA) rejected an approval, he believes. Colosimo is a member of an independent panel that occasionally advises Ema on neurological treatments.
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Biogen boss Viehbacher, on the other hand, sees a benefit for those affected: “What interests the patients are questions such as: Can I continue to dress myself? Can I eat on my own? Can I keep living at home?” he said. This was also examined in the studies and an improvement of 37 percent was seen compared to the placebo group.
“We have developed a therapy that is effective,” says the pharmaceutical manager. Now there is a real incentive for researchers to show what else they can do. “You usually don’t get a perfect drug on the first try,” said the Biogen boss.
Biogen needs a new growth driver
Analysts expect that the Ema will approve the drug, but the majority of sales with lecanemab will probably be made in the USA. Barclays experts expect sales of 2.6 billion dollars in Europe and 3.7 billion dollars in the USA by 2032. The analysts from Bloomberg Intelligence assume only two billion dollars in the USA.
In any case, Viehbacher wants to make the drug an important growth driver, which the company urgently needs, with sales of around ten billion dollars recently. Because in the most important business area, therapies against multiple sclerosis, sales are shrinking significantly due to growing competition, including copycat drugs.
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