Frankfurt On Friday evening, the American drug agency FDA granted preliminary approval for the new Alzheimer’s drug lecanemab, thereby raising hopes for further progress in the fight against dementia.
The Japanese company Eisai and the US biotech group Biogen, who jointly developed the active ingredient, now want to bring the drug onto the US market under the name Leqembi at annual therapy costs of $26,500 and also apply for approval in Europe in the next few months. Eisai’s stock gained around 6 percent in US trading after the FDA’s announcement, and Biogen’s share price rose around 4 percent.
The active ingredient lecanemab is considered an important milestone in the development of Alzheimer’s drugs. However, the remedy is still far from curing the disease and is also associated with some side effects. At the same time, however, it is the first active substance that was able to show in a clinical study relevant for approval that it can slow down the progression of Alzheimer’s disease.
The FDA therefore spoke of “significant progress in the fight for effective treatment of Alzheimer’s disease.” As Eisai and Biogen announced in September, lecanemab was able to reverse the cognitive decline in the treated patients compared to the comparison group in a large phase 3 study 27 percent reduction. Half of the approximately 1,800 study participants received either lecanemab or a dummy drug.
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The drug was administered as an infusion over a period of 18 months at two-week intervals. The mental abilities of the participants were recorded using the Clinical Dementia Ratings (CDR), an established system for measuring dementia symptoms.
The provisional approval that has now been granted is based on a previous smaller study. However, Eisai and Biogen also want to apply for regular approval on the basis of the larger study that has now been published.
Bump for the amyloid hypothesis
Lecanemab is an antibody that is directed against certain protein deposits in the brain cells, so-called beta-amyloids. According to experts, it also represents the first significant confirmation of the recently rather controversial amyloid hypothesis. It states that Alzheimer’s is triggered by amyloids and that removing this deposit with the help of medication can counteract the damage to brain cells.
Numerous tested active ingredients based on this concept had previously failed in clinical studies. The most recent example is the active ingredient gantenerumab, which the Basel-based Roche group developed in cooperation with Morphosys. The drug Aducanumab, also developed by Biogen and Eisai, was approved by the FDA in 2020 against the advice of a panel of experts, but the clinical studies in this case did not provide statistically clear results either.
US health insurance companies therefore refused reimbursement for the drug, and in Europe Biogen had to withdraw the application for approval. The controversial drug, which Biogen and Eisai initially offered for $56,000 a year, turned out to be a commercial flop for the two companies.
With lecanemab they now get a second chance. In this case, the clinical test results are considered to be significantly more valid and meaningful. Jörg Schulz, Director of the Clinic for Neurology at RWTH Aachen University, spoke in the run-up to the FDA decision of data that were “consistent and convincing across all endpoints and test results”. The Munich medical professor and head of the department for neurodegenerative diseases at the Ludwig-Maximilians-University, Christian Haas, assessed the test results as a “turning point”. In principle, they gave hope for approval as a drug.
The FDA called the drug “a significant advance in the fight to effectively treat Alzheimer’s disease.”
(Photo: AP)
Theoretically, there is enormous market potential for successful Alzheimer’s drugs. According to estimates by the WHO, around 55 million people worldwide are affected by dementia, around two thirds of whom are likely to suffer from Alzheimer’s. In the United States, the disease is the sixth leading cause of death. The number of Alzheimer’s patients there is estimated at around 6.5 million, in Germany at more than 1.5 million.
The new development from Eisai and Biogen will initially only be suitable for a fraction of Alzheimer’s patients. Because the drug was only tested in patients in the early stages of the disease and, according to the FDA, it should initially only be possible to prescribe it for such patients. In addition, it is not yet clear under what conditions the American state insurers will reimburse the costs. Current rules state that they only reimburse Alzheimer’s drugs if they were prescribed as part of clinical trials.
Discussions about the risk-benefit balance
In the case of the active ingredient lecanemab, there should also be intense debates about how much medical benefit the drug really offers. The treatment must be accompanied by relatively intensive accompanying diagnostics, as similar to other amyloid blockers, cases of swelling and microbleeding in the brain have been observed relatively frequently during treatment.
The discussions about the risk-benefit ratio were also fueled by three deaths that may be related to the active substance. They suggest that the drug, in combination with common blood thinners, could pose an increased risk of dangerous bleeding.
Irrespective of this, analysts see great potential in the product given the enormous need for therapy for Alzheimer’s. The British analysis company Evaluate Pharma, which aggregates market data and analyst estimates, sees the drug as potentially the most profitable new launch of the current year. By 2028, annual sales could then increase to three billion dollars.
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