The FDA granted accelerated approval to lecanemab – a new Alzheimer’s drug – on Jan. 6, despite recent disputes.
Lecanemab is designed to bind to the protein amyloid beta – a harmful protein mostly found in the brains of Alzheimer’s patients – helping to remove the protein in the early stages of the disease to lower its impact. The drug proved to lower cognitive decline by 27% over the course of an 18-month study.
“The approval for lecanemab, a new treatment for people living with mild cognitive impairment and early dementia due to AD – an irreversible, degenerative and fatal brain disease – builds on the promising data published in the New England Journal of Medicine and validated by the views of over 140 physicians who recently sent a letter to the FDA supporting the approval,” Sue Peschin, MHS, president and CEO of the Alliance for Aging Research, said in a press release.
In 2021, the FDA approved Aduhelm, a similar drug by the same creator. After a recent investigation, House committees found that the FDA overruled scientific advisors in their decision to approve the drug, which showed little effectiveness in early studies.
However, researchers say they believe lecanemab deserves its recent approval.
“We believe, based on the totality of positive data from clinical trials of this treatment, that the FDA should approve,” Maria Carrillo, chief science officer for the Alzheimer’s Association, said in a statement days before the FDA decision. “Peer-reviewed, published results show lecanemab will provide patients in the earliest stages of Alzheimer’s more time to participate in daily life and live independently. It could mean many months more of recognizing their spouse, children and grandchildren.”
Although lecanemab trials did show evidence of reduced cognitive decline, there were two hemorrhage-related deaths throughout the three studies, which raised concern about the drug’s safety.
Biogen, the maker of the drug, reported that some participants experienced infusion reactions, microhemorrhages, headaches and falls throughout the trials.
Still, experts stand by the safety of the drug.
“We are closer than ever to developing a new generation of drugs that targets the many causes of this devastating disease,” Howard Fillit, MD, co-founder and chief science officer for the Alzheimer’s Drug Discovery Foundation said in a press release. “While several media reports have called attention to safety concerns, the data shows lecanemab was generally well-tolerated. It will require close monitoring, particularly in patients at higher risk, such as those taking [blood thinners], but overall, I see these results as starting us down a promising path.”
Because the drug is under accelerated approval, it is more likely to win full approval faster.
“Under the accelerated route, a drug can win earlier approval if it shows an effect on a ‘surrogate endpoint’ – in this case, amyloid plaque formation in the brain – that is reasonably likely to predict a clinical benefit to patients,” Carrillo explained in the release. “The use of the AAP has been an important regulatory mechanism for the FDA to allow for earlier approval of drugs that treat serious and life-threatening illnesses than would occur through the traditional approval program.”
If lecanemab makes it to full approvals, the next step will be for Medicare to approve coverage of the drug, which it refused to do for Aduhelm.
“The Alliance calls on [Medicare Services (CMS)] to rescind its current extreme barriers to coverage for anti-amyloid therapies,” Carrillo said. “As the predominant insurer for individuals with Alzheimer’s disease, it is imperative that CMS provide immediate national coverage for Medicare beneficiaries.”