EMA Agrees to Review Biogen, Eisai Request for Aducanumab’s Approval

The European Medicines Agency (EMA) has agreed to review Biogen and Eisai’s application requesting the approval of aducanumab (BIIB037) for the treatment of Alzheimer’s disease.

The companies’ approval request — in the form of a marketing authorization application (MAA) — will be analyzed by the EMA following the usual review timetable for new medications, which can take up to 210 days. If approved, the investigational treatment will become the first therapy for Alzheimer’s that has the potential to lower patients’ clinical decline and alter the course of the disease.

“Alzheimer’s disease has become a significant and growing burden for societies around the world, and we believe aducanumab represents the first breakthrough that can change the course of this devastating disease,” Michel Vounatsos, CEO of Biogen, said in a press release.

“We are committed to working with regulatory authorities worldwide and we look forward to the European Medicines Agency’s review of this application,” Vounatsos added.

Developed by Biogen in collaboration with Eisai, aducanumab is a man-made antibody that is designed to bind to the toxic clumps of beta-amyloid protein that are thought to be the underlying cause of neurodegeneration in Alzheimer’s.

Earlier this year, the companies submitted a similar request for approval — in the form of a biologics license application — to the U.S. Food and Drug Administration (FDA). The agency is reviewing aducanumab’s application under priority review, and has set an action date of March 7, 2021, to come to a final decision on whether to approve the therapy to treat Alzheimer’s patients.

However, a Nov. 6 meeting cast a future FDA approval in doubt as an advisory committee suggested that current data from trials are not sufficient to prove aducanumab’s effectiveness. Advisory committees provide recommendations for the FDA to consider in reviewing potential therapies, and the agency will continue to review aducanumab ahead of the March 7 deadline.

Aducanumab’s approval requests are backed by clinical data showing that the experimental therapy was able to lower the number of amyloid plaques and slow the progression of cognitive decline in patients with early Alzheimer’s who had mild cognitive impairments associated with the disease and mild Alzheimer’s dementia.

In a Phase 3 trial called EMERGE (NCT02484547), aducanumab was able to slow the progression of cognitive and functional impairments — assessed by the Clinical Dementia Rating-Sum of Boxes score — by 23% within 78 weeks when given at the highest dose, compared with a placebo.

The investigational therapy also lowered the scores of other measures of clinical disease progression, including the Mini-Mental State Examination and the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 13 Items, as well as the buildup of amyloid plaques in patients’ brain.

These findings also were supported by data from another Phase 3 trial, called ENGAGE (NCT02477800), which showed aducanumab was associated with significant benefits in a subgroup of patients who had sufficient exposure to the high dose of the therapy.

Similar benefits in slowing cognitive decline and the accumulation of toxic deposits of beta-amyloid also were reported in a Phase 1b trial (NCT01677572), known as PRIME, which assessed the safety and tolerability of multiple doses of the therapy in patients with early Alzheimer’s.

“There are no treatments available that impact the progression of Alzheimer’s disease by addressing the underlying disease pathology. The potential that aducanumab may hold to effectively reduce the clinical decline brings new hope to people and families living with this devastating disease,” said Haruo Naito, CEO of Eisai.

“The acceptance of the Marketing Authorization Application in the European Union is an important milestone as we work towards making this potential treatment available around the world,” Naito added.