Hope for Alzheimer's patients: A drug has been approved for the first time to treat an underlying cause of the nervous disease.
The US health authority FDA gave the drug aducanumab from the American biotech company Biogen the green light on Monday.
Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research, said the data from the regulatory filing is very complex and there is still some uncertainty about the drug's clinical benefit.
However, the authority had come to the conclusion that the benefits of the agent outweigh the risks.
With the approval, Alzheimer's patients have "an important and new treatment for combating this disease".
Sales potential of $ 10 billion
The agent had initially achieved positive results in a clinical study, but these were not confirmed in another study.
An FDA advisory panel voted against the drug in November.
The authority is not obliged to listen to the recommendations of the panel of experts, but usually follows them.
Guggenheim analyst Yatin Suneja had previously stated that approval would likely depend on the willingness of the FDA to approve a drug on the basis of relatively weak data for a disease that affects millions and offers few current treatment options.
“This is good news for Alzheimer's patients,” said Alzheimer's specialist Ronald Petersen from the Mayo Clinic, speaking of a “special day”. However, he pointed out that the remedy did not bring any cure. "There is hope that this will slow the progression of the disease." Biogen can count on a billion dollar business: Guggenheim analyst Suneja believes the drug will have peak sales potential of at least ten billion dollars.
Aducanumab aims to clear beta-amyloid - one of the two characteristic protein deposits of Alzheimer's - from the brains of patients at an early stage of the disease.
According to the FDA, clinical trials of the compound, to be marketed under the name Aduhelm, showed a significant reduction in deposits.
However, some patients developed potentially dangerous brain swelling.
The FDA expects Biogen to conduct another clinical trial after approval to evaluate the drug's usefulness.
If the drug does not work as intended, the agency could take it off the market, the FDA emphasized.
So far there is no effective therapy for the rapidly progressing dementia, in which protein clumps accumulate in the brain of those affected and damage the nerve cells. Numerous pharmaceutical companies have already had to cope with failures in this research field. Current medications can only relieve symptoms.