It’s official: Roughly half a year after the FDA granted an accelerated nod to Eisai and Biogen’s new Alzheimer’s disease med lecanemab, the companies have converted that green light into a full-fledged, traditional approval. Now, the drug—also known as Leqembi—is set to unshackle itself from a strict coverage determination by the Centers for Medicare & Medicaid services (CMS).
With a full approval in hand, Leqembi has become the first treatment shown to reduce the rate of disease progression and slow cognitive and functional decline in adults with Alzheimer’s under the traditional approval pathway, Eisai executive vice president Alex Scott, who oversees government affairs for the company, said in an interview.
Those benefits stand across a “broad patient group that’s generalizable to the whole Medicare population,” he added.
Leqembi won an accelerated FDA approval in January based on its ability to reduce amyloid beta plaque, a marker of Alzheimer’s disease that's believed to be associated with improved outcomes.
For Thursday’s full nod, the partners submitted data showing the drug's benefit on an endpoint called the Clinical Dementia Rating-Sum of Boxes after 18 months of treatment. The scale rates the severity of dementia to diagnose and assess a patient's condition.
The approval comes after an FDA committee of outside experts voted unanimously that Leqembi’s pivotal trial results verify the med’s clinical benefit, with panel members describing the trial data as “robust,” “meaningful,” “consistent” and “significant.”
Overall, Eisai is “very pleased” with Leqembi’s label, Steven Hersch, VP of clinical research for new modalities and neurology and the international project lead for Leqembi, added in the interview.
First, the label portrays the data from Leqembi’s phase 3 Clarity AD trial, Hersch explained. Second, the approved indication is “pretty similar to the indication that was in the accelerated approval," the exec said.
In addition, Hersch said there are virtually “no limitations on prescribing" Leqembi to Alzheimer's patients noted in the label.
Safety and the risk of side effects known as amyloid-related imaging abnormalities, or ARIA, form another important discussion topic around Leqembi, and those concerns have culminated in a box warning.
“That box warning is an important way to signal to prescribers that it’s important that they understand ARIA and important that they be able to educate and communicate with patients and caregivers about it so that they can have very informed discussions about risk and benefit,” Hersch said.
Based on the language in the label—which suggests ARIA rates and timing may vary between products—that box warning will likely apply to all future Alzheimer’s drugs in the anti-amyloid class, Scott added.
Even still, “the rates of ARIA for our product are actually quite low,” Hersch pointed out.
With that in mind, it will remain important for neurologists and other prescribers to learn more about ARIA “than they’ve needed to in the past,” Hersch explained.
Launching in earnest
Beyond the simple regulatory designation of boasting a full approval, this development carries practical importance for the Leqembi launch. That's because the CMS has promised wider availability of Alzheimer's drugs approved under the FDA's traditional pathway.
Early last month, the agency said it would cover Alzheimer's drugs under a registry approach in a bid to continue collecting information as launches progress.
CMS has suggested that upon traditional approval, its portal-based registry for Leqembi will be up and available for utilization on the very same day, Scott said, adding, “we’ve been in extensive conversations with CMS over the last 18 months.”
Prior to the drug’s full approval, Leqembi—plus any new Alzheimer’s treatment carrying an accelerated approval—could only be covered in clinical trial settings to facilitate ongoing collection of data.
“We don’t have any special insight in terms of exactly what this [coverage] looks like, but we’re hopeful that it will be this light touch, easy-to-use registry that only needs to collect items that are already in a patient’s medical record,” he said.
Following Eisai and Biogen’s approval Thursday evening, CMS released a consumer factsheet telegraphing its plans to cover the drug “more broadly.” Medicare noted it will act quickly by implementing a new billing code for Leqembi on a “very compressed schedule.” As for the registry, which opened immediately after Leqembi’s approval, analysts at Mizuho Securities called the form “very reasonable.”
Now, Eisai is scaling up its field-based organizations—from sales reps and medical science liaisons to access reimbursement agents, Scott said. That process is “well underway” with a “fair number” of staffers already deployed, according to Scott.
He added that the company also has a wealth of educational materials it’s assembling.
“This is not a fast build,” Scott stressed. “This is a slow and careful build that we’re being very conscious about.”
As for other ways Eisai is working to improve patient experience, Hersch pointed to the company’s development of a subcutaneous version of Leqembi, which could allow for administration outside of infusion centers. Eisai is also working on a maintenance dose regimen that could permit less frequent dosing, he said.
Leqembi is Biogen and Eisai's second Alzheimer's drug after their failed first offering Aduhelm. That drug won an accelerated FDA approval in 2021 and never gained market traction due to skepticism around its data and the CMS coverage restriction.
With the full approval of Leqembi, Scott said he hopes “a lot of the controversy should now get past us,” allowing the company to focus on serving patients.