- After almost 20 years of no new treatments, Biogen has received accelerated approval for aducanumab, also known as Aduhelm. This medication treats Alzheimer’s by addressing the protein plaques associated with the illness.
- Clinical trials have shown that the medication slows disease development in the early stages by helping in the removal of plaques associated with the disease.
- However, the approval of the drug for Alzheimer’s is contentious because there is no clear evidence that it improves symptoms.
The Food and Drug Administration (F.D.A.) announced accelerated approval for Biogen’s Aduhelm, the brand name for aducanumab. Despite disagreement about whether clinical evidence indicates it works, it is the first medication to treat a potential cause of Alzheimer’s disease.
Other medicines relieved some symptoms. But, one clinical study found that Aduhelm slows the progression of plaques associated with Alzheimer’s in individuals in the early stages of the illness.
However, this drug for Alzheimer’s is also controversial. This is because it is unclear how effective it will treat its clinical symptoms.
How does the drug for Alzheimer’s work, exactly?
Aducanumab is an amyloid beta-directed antibody. This means it targets amyloid plaques present in Alzheimer’s patient’s brains. According to the National Institute on Aging, these plaques are made of harmful proteins that collect between neurons and impair cell function (N.I.A.).
Researchers give Aducanumab as an IV injection once a month. It is the first FDA-approved dementia therapy that targets the disease’s process rather than just its symptoms. In addition, the clinical trials for Aduhelm were the first to show that a decrease in these plaques reduces this terrible form of dementia.
But what does the data say about aducanumab?
Researchers tested Aducanumab in two Phase-3 clinical trials. They included 3,285 people with early Alzheimer’s disease at 348 sites in 20 countries. One research found that patients’ mental decline was reduced. But the second experiment did not. However, in all studies, Aduhelm consistently decreased the number of amyloid plaques in the brain in a dose- and time-dependent fashion.
However, the road to approval for aducanumab has not been easy. Biogen stated in March 2019 that it was discontinuing the medicine’s ENGAGE and EMERGE studies after an analysis showed that the trials were unlikely to meet their objectives and that the drug for Alzheimer’s no longer had a possibility of benefitting patients.
Researchers released new data from 318 study participants who completed the trials three months later, with positive results. Months later, Biogen announced that aducanumab is really effective, citing results from the EMERGE trial. It found that patients who got a high dose of aducanumab (6 or 10 mg/kg) performed better on cognitive function tests than those who received a placebo. However, in the ENGAGE trial, there were no changes between individuals who got aducanumab and those who received a placebo.
Why is aducanumab so controversial?
Every first medication of any kind is studied more by doctors and scientists than subsequent drugs.
However, there are several reasons why some scientists and clinicians are wary about the medication:
1. Aducanumab has potential risks and adverse effects
According to Biogen, aducanumab might cause significant adverse effects. These include temporary swelling and bleeding in the brain. According to Biogen, most people will not have any symptoms. But some may experience a headache, confusion, dizziness, visual abnormalities, and nausea. As a result, the company advises patients to have M.R.I. scans before and during treatment.
According to Amit Sachdev, M.D., the risks of brain swelling are “serious” and can potentially be deadly. So constant monitoring is really important.
Biogen also states that some patients have experienced allergic reactions to the drug. These include swelling of the face, lips, mouth, or tongue, as well as hives. It will be important to assess if the advantages outside of a clinical trial environment exceed these [potential] side effects.
2. It is unclear whether aducanumab is effective
Once again, Biogen stopped two Phase-3 clinical studies of the drug for Alzheimer’s after an analysis revealed that it was unlikely to benefit patients. Even after more data became available, just one study shows that aducanumab beat a placebo. Finally, there is conflicting data from clinical trials as to whether or not it works.
3. It’s expensive
Biogen announced this week in an investor press release that the drug will cost $56,000 per year. The company state that “the out-of-pocket cost” for consumers with insurance would vary based on their coverage. Regular M.R.I.s during therapy would be an additional cost as well.
According to Dr. Sachdev, the price is simply much too high. I would have liked to see the pricing trend for developing dementia drugs follow that of new migraine medications, which use very similar technology and cost much less, he says. In comparison, a carton of 10 ampules of the injectable migraine medicine D.H.E 45 costs $11,767.
Biogen will conduct a post-approval clinical study
Even with accelerated permission, Biogen will conduct more study on Aduhelm.
According to the F.D.A., this type of approval can be based on a medication’s effect on a surrogate endpoint that is reasonably likely to predict a therapeutic benefit to patients. Still, it needs a post-approval trial to verify that the treatment provides the expected clinical benefit.
These are known as phase 4 confirmatory studies. And they are designed to show that a treatment has clinical benefits. If this trial fails to show effectiveness, the F.D.A. may reconsider its decision.
Negative findings might lead to approval withdrawal. Phase 4 studies allow for more data collection.
According to the Alzheimer’s Association, Aduhelm is only one part of the therapy
This therapy will spark the interest of many. Still, it is not the only critical component of Alzheimer’s treatment and care, said Joanne Pike, DrPH, chief strategy officer of the Alzheimer’s Association, in an emailed statement.
The Alzheimer’s Association has made it a priority to collaborate with healthcare systems, clinicians, and the Centers for Medicare and Medicaid Services to guarantee early and accurate diagnosis, as well as access to treatment, care management, and care planning, she continued.
Pike also said that the association would do all in its power to guarantee that everyone who would benefit from the medicine and FDA-required diagnostic tests had access to it.
The bottom line
After nearly two decades of no new treatments, biotech company Biogen has won accelerated approval for Aduhelm, the brand name for aducanumab. It is a medication that treats Alzheimer’s by attacking the protein plaques associated with the illness.
Multiple clinical trials, according to Biogen, have proven that the medication slows disease development in its early phases. However, its approval is controversial because the medication does not clearly show that it helps symptoms.
According to experts, medication is only one aspect of Alzheimer’s treatment. Early diagnosis, access to treatment, care management, and care planning are all important aspects of caring for people with the disease.
