Biogen has filed for U.S. approval for its investigational Alzheimer’s disease treatment aducanumab, which may become the world’s first treatment for the disease.

The company said it has submitted a Biologics License Application (BLA) to the Food and Drug Administration on Wednesday.

The submitted documents included not only phase-3 EMERGE and ENGAGE trials on aducanumab but phase-1b PRIME data. The company also asked the FDA for priority review.

Within the next 60 days, the FDA has to decide whether to accept Biogen’s BLA for aducanumab. If it agrees to it, it has to decide whether to conduct a priority review on the drug.

“Alzheimer’s disease remains one of the greatest public health challenges of our time. It robs memories, independence, and eventually the ability to perform basic tasks from the people we love,” said Biogen CEO Michel Vounatsos in a statement.

Vounatsos said the aducanumab BLA is the first filing for FDA approval of a treatment that addresses the clinical decline associated with the illness, as well as the pathology of the disease. “We are committed to driving progress for the Alzheimer’s disease community and look forward to the FDA’s review of our filing,” he added.

The phase-3 EMERGE and ENGAGE trials evaluated the efficacy of aducanumab compared with placebo in 3,285 patients with early-stage Alzheimer’s disease registered at 348 institutions in 20 countries for 18 months.

The primary endpoint was set as changes in the clinical dementia rating-sum of boxes (CDR-SB) score. Secondary objectives were to evaluate the mini-mental state examination (MMSE), Alzheimer’s disease assessment scale-cognitive subscale 13 items (ADAS-Cog 13), and Alzheimer’s disease cooperative study-activities of daily living inventory mild cognitive impairment version (ADCS-ADL-MCI).

Aducanumab met all the primary and secondary endpoints in the EMERGE study. However, in the ENGAGE trial, the drug showed efficacy only in some high dose groups, and experts are split over the ENGAGE study outcome.

Pharmaceutical industry officials are paying extra attention to whether the FDA will accept Biogen’s license application for aducanumab.

kym@docdocdoc.co.kr

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