FDA approves new Alzheimer's drug, Aduhelm, the first in nearly two decades

The Food and Drug Administration on Monday approved a high-profile Alzheimer’s drug, a controversial decision to green light the first new drug to slow the mind-robbing disease in nearly two decades.

The FDA’s landmark approval of Biogen’s aducanumab will require the drug company to conduct a post-approval study to monitor its effectiveness. The company said it will market the drug under the name Aduhelm.

The FDA’s approval comes despite a contradictory recommendation from experts on the agency’s Peripheral and Central Nervous System Drugs Advisory Committee, which last November cited conflicting results from two main clinical trials. The FDA, however, decided the drug may provide “meaningful therapeutic benefit over existing treatments,” Dr. Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research, said in a statement.

The FDA granted accelerated approval of the drug, a standard to provide earlier access to patients with serious diseases with an unmet need. Despite “residual uncertainty” about the drug’s benefit, Cavazzoni said, the agency concluded the drug met the agency’s standard as potential benefits outweighed the risks.

Alzheimer’s patient advocacy organizations praised the FDA’s decision to approve the drug, which is administered by IV once every four weeks to slow decline in people with mild memory and thinking problems. Biogen said the list price would be $4,312 per treatment for a person of average weight – a price that would total about $56,000 per year, before insurance.

Harry Johns, Alzheimer’s Association president and chief executive officer, said his organization will focus on ensuring eligible patients have access to the drug and Medicare and private insurers cover testing and treatment.

Johns said the approval is the culmination of years of efforts to fund research of new treatments to slow the deadly march of Alzheimer’s and other forms of dementia. The group estimates about 6.2 million Americans are afflicted with Alzheimer’s.

“It really is a new day for all of our constituents, so many who already have lost family members, so many who face Alzheimer’s today,” said Johns. “I want to make clear this is not a cure. As in the case of so many other diseases, the first treatments are imperfect, but they can and do make a difference and that is very meaningful to those families.”

Still unanswered are thorny questions about how doctors determine if a patient is eligible for the drug and whether patients will need to get expensive PET scans to detect amyloid beta plaque deposits in their brain.

“It’s going to be months before we know if anybody is going to pay for medication, the infusions, the MRIs, the PET scans that will be needed,” said Dr. Pierre Tariot, director of the director of Banner Alzheimer’s Institute in Phoenix. “Who will be trained to disclose the amyloid information to the patient and the families? There’s a lot that has to be done.”

The drug is a monoclonal antibody that works by removing amyloid beta from the brain. Scientists have long theorized plaques formed by these protein deposits in the brains of Alzheimer’s patients kill brain cells and lead to cognitive decline. However, drug companies have invested billions on drugs that target or remove amyloid but have not yet proven the drugs slow memory and thinking problems.

The FDA said the approval of Aduhelm is based on a “surrogate endpoint” that the drug effectively removed amyloid plaque from the brain. Such an endpoint predicts but does not measure whether the drug will slow memory or thinking decline.

Biogen will be required to conduct a post-approval study to verify the drug works and monitor for known side effects such as brain swelling. If the study does not verify the drug’s clinical benefit, the FDA could remove the drug from the market.

In a document released last November, FDA reviewers gave a mostly positive assessment of the drug, finding “substantial evidence of effectiveness” as a treatment for Alzheimer’s disease. However, in the same document, another reviewer raised questions about statistical methods, was not convinced of the evidence and recommended another trial.

Biogen CEO Michel Vounatsos said the authorization follows more than a decade of groundbreaking research of Alzheimer’s disease.

“We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come,” Vounatsos said. “We are grateful for the contributions of thousands of patients and caregivers who participated in our clinical trials, as well as for the dedication of our scientists and researchers.”

Aduhelm has stirred considerable debate among Alzheimer’s researchers for its unconventional path to approval.

After a small, initial study showed the monoclonal antibody was safe, Biogen launched two large trials in 2015 to gauge the effectiveness of the drug, which removes beta amyloid plaques from the brain of an Alzheimer’s patient.

In March 2019, the company halted companion studies that produced different results after an independent “futility analysis” concluded the drug did not seem to work. At the time, it appeared to join a growing list of amyloid-targeting drugs over the past two decades that looked promising in early studies only to fizzle in late-stage, expensive trials.

But after reviewing data from the two trials, Biogen decided there was enough evidence from one trial with positive results and signals from a subgroup of the second trial to support high doses of the drug as safe and effective. The company held talks with the FDA and submitted a new drug application, which the agency fast-tracked with priority review.

Dr. Howard Fillit, founding executive director and chief science officer at the Alzheimer’s Drug Discovery Foundation, said the FDA struck the right balance of giving patients access to a drug while requiring ongoing monitoring through a post-approval study. Had the FDA mandated another clinical trial before approval, it would have taken three to five years and a significant investment Biogen might not be willing to underwrite.

“Part of the target is for people who have a serious disease that is ultimately fatal,” Fillit said. “Finally, the FDA is recognizing that Alzheimer’s disease is a serious disease. Right now, there are no survivors from Alzheimer’s disease.”

Ken Alltucker is on Twitter at @kalltucker, or can be emailed at alltuck@usatoday.com

News Related


Mum reveals $10 reason son can't have 'normal life'

For Lykera Parker, seeing another child walk can trigger a pang of sadness. Because she knows that while her son Kalarny is as bubbly and outgoing as any four-year-old, he ... Read more »

COVID-19: Cases fall in the Netherlands and UK, while Italy and Romania push vaccines

While COVID-19 cases fall in some European countries, others fear a fourth wave of infections is underway. Cases in the Netherlands have fallen by 44% compared to last week, according ... Read more »

Schools implement mask mandates even as some states impose bans; US reports one case every second: Live COVID-19 updates

The U.S. Department of Education Monday released a roadmap for the return to school this fall, encouraging districts to invest in physical and emotional support for students. Among the roadmap’s ... Read more »

Cobblestone stones honor WWII gay and lesbian resistance fighters

Karel Pekelharing stone. Photo: DutchNews.nl Nine Jewish gay and lesbian resistance figurines are to be given their own “Stolperstein” or commemorative street tiles in front of their last known address. ... Read more »

Covid UK news – live: Vaccine to be offered to 16 and 17-year-olds as UK sees highest deaths since mid-March

✕ Close Coronavirus in numbers The coronavirus vaccination programme looks set to be rolled out to more than a million 16- and 17-year-olds. It is expected ministers will on Wednesday ... Read more »

Obese mice lose weight ‘sweating out' fat in an immune system experiment, despite eating more

In search of better treatments for type 2 diabetes and other consequences of obesity, Taku Kambayashi has long wondered if he could harness a bodily function that most think about ... Read more »

NYC will have the first government-issued vaccine mandate in the US. Here's what we know.

NYC Mayor Bill de Blasio just mandated proof of vaccination for indoor restaurants, gyms and performance venues. Here’s how that will work. Read more »

Peak hospital admissions seems to have been reached, 44% fewer new infections

Increase in IC In the past week, 541 new COVID-19 hospitalizations were registered compared to 564 the week before. “In the past week, there were 130 new admissions to the ... Read more »

The Path to Forgiveness When Someone Hurts You: How to Forgive, Let Go, and Protect Yourself

When you’ve been hurt by someone you trust, you might feel angry or resentful, especially if it keeps happening. Forgiving the person who is hurting you can rid you of ... Read more »

How to Boost Your Confidence (13 Steps to Help Your Social Anxiety)

Are you sometimes nervous to speak up and say what’s on your mind? It’s okay to be a little shy every now and then, but it might prevent you from ... Read more »

This South Korean company has figured out how to get the best night's sleep. Does sleep tech work?

A lack of sleep not only makes us grumpy in the mornings but it can also affect our health too. There are a range of gadgets out there with many ... Read more »

NHS must work in “new ways” to deliver care, says new chief executive

Amanda Pritchard during a visit to University College Hospital London, following the announcement of her appointment as the new chief executive of the NHS in England. (PA) The health service ... Read more »

3 ways to show you've gotten the COVID-19 vaccine

Did you misplace your vaccine passport? Here are three other ways you can prove you’ve been vaccinated. Read more »

Covid-19 antibody test: what does your result mean? Why a high score is good but doesn't tell the whole story

Standing in a taxi queue recently I overheard a middle-aged man say to a woman of similar age ahead of him in line, “I got 650”. Whatever he was referring ... Read more »

One-third of all COVID cases reported in Florida and Texas; US reaches 70% of partially vaccinated adults: COVID-19 updates

Florida and Texas had one-third of all COVID cases reported last week, White House COVID-19 Response coordinator Jeff Zients said during a Monday news conference. Florida broke two records — ... Read more »

Rabobank to compensate customers who have paid too much interest

Photo: Rabobank Rabobank is working to settle thousands of customers who have paid large amounts of interest on consumer loans, Cooperative Bank said Tuesday. The decision focuses on the high ... Read more »

Covid UK news - live: PM U-turns on amber watchlist as England could be ‘over the edge' of third wave

✕ Close Boris Johnson hints at U-turn on amber watchlist Plans for an “amber watchlist” have been abandoned as ministers prepare to review the traffic light system which will decide ... Read more »

How to Verify a Physician's License in California

Before you choose a physician for any type of treatment, it’s always a good idea to verify their license. This assures you that their license is in good standing and ... Read more »

Sailing Bronze has won a total of 19 Dutch medals and will win even more.

Annemiek Bekkering and Annette Duetz during the race. Photo: Olaf Kraak ANP In the disappointing medal race of Sailor Annemiek Bekkering and Annette Duetz, she was pushed back to the ... Read more »

From AI to ice vests: How Tokyo 2020 athletes are adapting to the hottest Olympics ever

Tokyo 2020 is likely to go down in history as the hottest modern Olympic and Paralympic Games ever. Daily high temperatures in the Japanese capital have hovered around 31C throughout ... Read more »

How to Use a Monocular

A monocular is like a small telescope. It is smaller and lighter than binoculars while packing the same power. To use a monocular, make sure that you keep a firm ... Read more »

Poorer communities see twice as many smoking related cancers as richer areas, says study

Smoking is linked to poverty and is causing more cancers in poorer communities (Getty Images/iStockphoto) There are nearly twice as many cancers caused by smoking among the poorest people in ... Read more »

Paramedics left in tears from ‘unsustainable demand', warns union

Unison has warned ambulance chiefs that the pressure is taking its toll on their workforce (Getty) Paramedics are being left in tears at the end of stressful shifts, with some ... Read more »

How to Use Apple Health

This wikiHow teaches you how to get started with Apple Health on your iPhone. The Health app makes it easy to access your important health information, including your medical history, ... Read more »

How to Understand Social Distancing

The term “social distancing” is all over the news, but what does it really mean? This term refers to staying a specific distance away from other people to help prevent ... Read more »

How to Treat a Bruised Thigh Muscle

Ouch! A bruised thigh is no joke. The front of your thigh is composed of large muscles called your quadriceps, and anybody who’s ever taken a direct hit there can ... Read more »

How to Treat Tunnel Wounds

A tunnel wound is a secondary wound that occurs alongside a primary wound, and it’s usually caused by an infection or pressure. This kind of wound extends into layers of ... Read more »

How to Treat Overmethylation

Methylation is a fairly complex process, but basically, it’s the natural process where methyl groups (one carbon and three hydrogen atoms) are transferred throughout your body. This happens billions of ... Read more »

How to Treat Numbness in Legs and Feet

Numbness in your legs and feet can cause discomfort and awkwardness, but it’s usually no cause for alarm. If you sit or stand in the same position for an extended ... Read more »

How to Treat Liver Fibrosis

Your liver is an incredible organ—it can grow back from almost nothing. But if it’s damaged too often, it can lead to a condition called fibrosis. The good news is ... Read more »

50 per cent vaxxed is not enough to safely lift restrictions: Doherty Institute

If NSW reaches its target of 6 million Covid-19 vaccinations by the end of the month it will still be “a long way” from being safe from the virus, according ... Read more »

How to Treat Crepitus

Crepitus is a general term for the crackling or popping sound made by a joint. This is typically caused by cartilage or soft tissue rubbing against a joint during movement, ... Read more »

How to Treat Cold Feet

Having cold feet is a pretty common problem, especially during the wintertime. However, if you have persistent cold feet, you could have an underlying condition such as Reynaud’s phenomenon. Fortunately, ... Read more »

How to Treat Buttock Folliculitis

Folliculitis is a common condition causing inflamed hair follicles and a raised rash. While it can occur anywhere on your body, the buttocks are a common area for an outbreak. ... Read more »

'People who do not want to be vaccinated may go elsewhere': Court backs Indiana University mandate

INDIANAPOLIS — Students who don’t like Indiana University’s COVID-19 vaccine requirement can go elsewhere for their education. That was the message delivered by the 7th Circuit Court of Appeals in ... Read more »

How to Take a Mankind Unwanted Kit

A Mankind Unwanted Kit is made up of 1 pill called mifepristone and 4 pills called misoprostol that your OB/GYN can give you. These are taken to terminate an unwanted ... Read more »

How to Take Control of Your Health

No matter what our existing health, financial, mental, economic, or social situation is, there are things we can each do to take (better) control of our own health. No one ... Read more »

How to Take Care of Yourself

Taking care of yourself can feel like a really big task, but it doesn’t have to be overwhelming. Learn to manage your physical, mental, and emotional health so that you ... Read more »

Japanese shadow painting master delivers hope, delight in Seoul

Read more »

How to Store Ascorbic Acid

Ascorbic acid, or ascorbate, is another name for vitamin C. Ascorbic acid helps your body grow and repair itself and is an essential nutrient. Unfortunately, it is also very delicate ... Read more »