Alzheimer's: Biogen to Ask FDA to Approve Aducanumab As First Treatment for Early Stage of Disease

A biotechnology company which says it has developed the first drug to treat Alzheimer's disease plans to ask for the medication to be approved for use in the U.S.

Following discussions with the U.S. Food and Drug Administration (FDA), Biogen said in a statement on Tuesday that it would ask the agency to review aducanumab in early 2020.

The announcement comes as 5.8 million Americans are living with the neurodegenerative condition with an unknown cause, and for which there is no treatment or cure.

Biogen said patients with early Alzheimer's who were given a high dose of the drug "experienced significant benefits on measures of cognition and function such as memory, orientation, and language."

The firm also said participants "experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping, and doing laundry, and independently traveling out of the home."

If the FDA gives the green light, aducanumab would become the first treatment to target clinical decline in those with Alzheimer's disease.

Aducanumab targets the beta amyloid protein. The approval of the drug would therefore also bolster the amyloid hypothesis that the build-up of the substance contributes to the development of Alzheimer's disease, Biogen suggested.

In March, an independent data monitoring committee advised Biogen to stop studies on the medication after what is known as a futility analysis showed it was unlikely to meet its primary aims. Biogen subsequently discontinued its two clinical trials, stressing at the time that the decision was not linked to safety concerns.

By abandoning its efforts, Biogen shocked the pharmaceutical industry and cast doubt on the amyloid hypothesis, according to Stat News, as a number of other experimental drugs targeting the protein had previously failed.

But in the months that followed, Biogen carried out more research using a larger dataset, which returned positive results.

Al Sandrock, head of research and development and chief medical officer at Biogen, explained to Stat News that the data used to make the futility analysis came from December. But the trials finished in March, and the new research was completed by June.

Sandrock said Biogen met with the FDA in June and on October 21 to discuss the potential of aducanumab. "I have to pinch myself because I almost don't believe it yet," he said.

Michel Vounatsos, chief executive of Biogen's chief executive said in a statement: "With such a devastating disease that affects tens of millions worldwide, today's announcement is truly heartening in the fight against Alzheimer's. This is the result of groundbreaking research and is a testament to Biogen's steadfast determination to follow the science and do the right thing for patients.

"We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer's disease and the potential implication of these results for similar approaches targeting amyloid beta," he said.

Lead author of the study Professor Anton Porsteinsson, director of the University of Rochester Alzheimer's Disease Care, Research and Education Program, commented in a statement that the findings provide "new hope for the medical community, the patients, and their families."

The Alzheimer's Association, a non-profit health organization, said in a statement that it finds the development encouraging.

"We eagerly anticipate reviewing a full report of the findings from these studies and participating in a robust discussion of data," it said.

"No stone can be left unturned," the statement continued. "We must advance all potential treatment avenues and also explore methods for combining these approaches.

"Alzheimer's and other dementias are complex, and their effective treatment and prevention will likely also be a complex—but achievable—task. All currently pursued treatments that are considered safe should be continued to determine their efficacy."

James Pickett, head of research at the U.K.-based charity Alzheimer's Society, commented in a statement: "This is hugely exciting news. After the trial being stopped earlier this year because it appeared not to work, further analysis suggests that it does benefit people with dementia in the earliest stages.

"We're waiting for further data but this could be the first new treatment for Alzheimer's disease in over 15 years, and as such, has the potential to be a transformative discovery."

This article has been updated with comment from the Alzheimer's Association and James Pickett.

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A biotech company says they have created a drug which could treat Alzheimer's, a condition which affects the memory. A stock image shows an older person holding a photo from their youth. Getty
Alzheimer's: Biogen to Ask FDA to Approve Aducanumab As First Treatment for Early Stage of Disease | Health