In a long-awaited decision, the Food and Drug Administration on Monday, June 7, approved Aducanumab as the first new medication for Alzheimer’s disease in nearly two decades and the first to treat the underlying pathology of the disease – not just its symptoms.
The drug, which will go by the brand name, Aduhelm, was approved by the F.D.A. under the agency’s accelerated approval pathway, which can be used for a drug that is meant for a serious life-threatening illness such as Alzheimer’s disease and provides a meaningful therapeutic advantage as compared to existing treatments for that same condition.
Under the accelerated approval provisions, which provide those with Alzheimer’s with earlier access to the treatment, the F.D.A. is requiring a new randomized, controlled clinical trial called a Phase 4 trial, to verify the drug’s clinical benefit. The results of this post-market study are intended to erase any uncertainties about the drug’s ultimate clinical benefit or can lead to the F.D.A.’s initiation of proceedings to withdraw approval of the drug.
“The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit,” the F.D.A.’s director of the Center for Drug Evaluation and Research, Dr. Patrizia Cavazzoni, wrote on the agency’s website. She further explained, however, that “there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit.”
Aducanumab is given through a monthly intravenous infusion and targets a protein, amyloid, that clumps into plaques in the brains of persons with Alzheimer’s disease and reduces the levels of amyloid. In clinical trails conducted by Biogen and Eisai, those receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while those in the control group of the studies had no reduction of amyloid beta plaque.
Although the clinical trials for aducanumab were conducted on those with mild cognitive impairment or early-stage Alzheimer’s whose brains contained higher than-normal levels of amyloid the F.D.A approval for the drug does not contain any restrictions. The “label” simply says that the drug is “for the treatment of Alzheimer’s disease.”
“We are very excited with today’s F.D.A. announcement about aducanumab, especially after years of disappointment in the results of other potential treatments and we were pleased to be part of the advocacy efforts to urge approval,” said Ken Zaentz, President and CEO of Alzheimer’s New Jersey. This could be a turning point for Alzheimer’s disease research as the approval of aducanumab will likely serve as a catalyst for investment in additional disease modifying treatments.”
“There is now hope that for many with Alzheimer’s disease in New Jersey and beyond they will have access to a therapy that will give them more time to enjoy independence and improved quality of life and we look forward to positive results from the Phase 4 study. While we anticipate that there will be great demand for aducanumab, as those with Alzheimer’s and their caregivers have waited so long for a potential disease modifying treatment, we also want to stress that aducanumab may not be right for everyone who currently has Alzheimer’s. We urge having those important conversations with medical professionals to help make this determination, said Zaentz.
At the same time, Alzheimer’s New Jersey will continue to support access and affordability for those who may benefit.