UPDATE 6: Biogen and Eisai receive U.S. FDA approvals for Alzheimer’s drugs. Apply for full approval

(Adds Eisai CEO comments, detail in para 7-9-10

Deena Beesley and Julie Steenhuysen

Jan 7, 2017 (Reuters) – The U.S. Food and Drug Administration approved lecanemab, an Alzheimer’s drug developed by Eisai Co Ltd and Biogen Inc to treat patients with the disease in its earliest stages.

Eisai, Biogen and Biogen both confirmed that the Japanese drugmaker has applied for FDA approval.

Leqembi will sell the drug. It is part of a group of treatments that aims to slow down the progression of neurodegenerative diseases by removing toxic beta amyloid clumps from the brain.

Nearly all of the previous experimental drugs that used the same approach have failed.

“Today’s news is incredibly important,” said Dr. Howard Fillit, chief science officer of the Alzheimer’s Drug Discovery Foundation. “Our years of research into what is arguably the most complex disease humans face is paying off and it gives us hope that we can make Alzheimer’s not just treatable, but preventable.”

Eisai claimed that the drug would go on sale at an annual cost of $26,500. Biogen shares rose 3% to $279.40 after they were halted.

According to the Japanese company, it plans to also apply for marketing authorization for Leqembi within Japan and the European Union by March 31, the end its business year. The hope is that the Japanese authority will approve this application by then.

Eisai projected that there would be around 100,000 eligible U.S. patients for the drug within three years. The number will continue to grow over time.

“Our assumption is the drug will be available to approximately 2.5 million patients worldwide by 2030,” Eisai CEO Haruo Nagato stated to analysts and reporters in Tokyo.

Without revealing any figures, he stated that although the drug won’t generate significant profit right away, the product will begin contributing to our profits in the second or third half of the second year.

Dr. Erik Musiek from Washington University, a Barnes-Jewish Hospital neurologist, stated that he was “pleasantly shocked” at the drug’s cost.

“Considering the current market and the lack of good treatment options for diseases other than those we have, I believe it’s reasonable to expect,” he stated.

There are a few factors that will limit patient access, including Medicare reimbursement restrictions. This is the U.S. government program for Americans 65 years and older. It covers approximately 90% of those likely to be eligible for Leqembi.

“Without Centers for Medicare & Medicaid Services (CMS) and insurance coverage … access for those who could benefit from the newly-approved treatment will only be available to those who can pay out-of-pocket,” the Alzheimer’s Association said in a statement.

Leqembi was granted FDA approval under the FDA’s expedited review process. This expedited pathway speeds up access to drugs because of their potential impact on biomarkers related to disease that can be used to predict clinical benefit.

“This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s instead of only treating the symptoms of the disease,” FDA neuroscience official Billy Dunn said in a statement.

CMS stated Friday that the current restrictions on drug coverage for drugs approved under the accelerated path could be reviewed based on ongoing reviews.

CMS stated that if the drug is approved by FDA, it will be covered in greater depth. Officials at Eisai have stated that the company will submit data from a successful clinical trial in 1,800 patients to support a full standard review for Leqembi.

The CMS decision was in large part a response to an Alzheimer’s treatment previously offered by Eisai or Biogen. Aducanumab (sold under the name Aduhelm) was approved in 2021. There is little evidence that it slows cognitive decline, and the FDA did not object to the claims of outside experts.

Aduhelm was originally priced by Biogen at $56,000 annually. Then, the price was halved. Due to limited acceptance and lack of insurance coverage, Aduhelm sales fell to $4.5 million within the first nine months.

Lecanemab can be used for patients suffering from mild cognitive impairment or early Alzheimer’s dementia. Doctors believe this is a small percentage of the 6 million Americans who suffer from the memory-robbing disease.

Patients will be required to have their amyloid deposits tested in order to receive treatment. This can be done either by brain imaging or a spinal tap. To monitor for brain swelling, patients will also need periodic MRI scans.

According to the medicine’s label, doctors must be cautious when prescribing blood clot prevention drugs to lecanemab patients. According to an autopsy report published this week, this could pose a risk to safety.

The large trial of lecanemab infusion was shown to slow the rate of cognitive decline among patients with early Alzheimer’s disease by 27% in comparison to a placebo. Brain swelling was reported in nearly 13% of the patients who received Leqembi.

Dr. Babak Tousi at the Cleveland Clinic is a neurogeriatrician and said that the approval will make “big differences” in the field, as it’s based on biomarkers, not just symptoms.

He said, “It’s going change how we make an Alzheimer’s diagnosis with more accuracy.”

Tousi acknowledged the drug’s likely modest benefits. “But, it’s a benefit we weren’t able to achieve before this approval.”

(Reporting by Deena Belasley in Los Angeles, Bhanvi Satija, in Bengaluru; additional reporting Jaiveer Shahat in Bengaluru and Yuka Okashi in Tokyo; Editing and editing by Bill Berkrot and David Gregorio. William Mallard.