Europe backtracks and recommends approving donanemab: a promising Alzheimer's drug

Eli Lilly today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the use of donanemab for the treatment of early symptomatic Alzheimer’s disease in adults with confirmed amyloid disease who are heterozygous or do not carry the apolipoprotein E ε4 allele ( ApoE4 ). The European Commission is expected to make a final regulatory decision on the marketing authorization for donanemab in the European Union in the coming months.

"This positive opinion marks an important milestone in our work to bring donanemab to the right patients across Europe," said Patrik Jonsson , executive vice president and president of Lilly International . " Donanemab can make a significant difference in the lives of people with early symptomatic Alzheimer's disease , and Lilly remains committed to advancing the science through ongoing clinical trials ."

Alzheimer's disease currently affects 6.9 million people in Europe , and this figure is expected to almost double by 2050 due to the increasing ageing of the population 1-2. Approximately one-third of people with mild cognitive impairment or mild dementia due to Alzheimer's disease progress to the next clinical stage of the disease within a year 3.

"These data give us an idea of the magnitude of a disease with both health and social consequences, given the extreme dependence of patients in advanced stages," recalls José Antonio Sacristán , medical director of Lilly Spain . "Opinions like this one from the CHMP open the door to a new era in which, for the first time, we can consider changing the course of the disease in its initial stages. To achieve this, it is essential to strengthen early diagnostic capabilities , access to biomarkers, and the formation of multidisciplinary teams within our healthcare system," he states.

The positive opinion was based primarily on data from the TRAILBLAZER-ALZ 2 trial, which demonstrated that donanemab significantly slows cognitive and functional decline and reduces the risk of progression to the next clinical stage of the disease, and on data from the TRAILBLAZER-ALZ 6 trial, which evaluated a titration of the dosage regimen. In TRAILBLAZER-ALZ 6, the titration of the dosage regimen significantly reduced the incidence of amyloid-related imaging abnormalities with edema/effusion (ARIA-E) compared with the original regimen used in TRAILBLAZER-ALZ 2 at 24 and 52 weeks, achieving similar levels of amyloid plaque clearance and P-tau reduction .

Amyloid-related imaging abnormalities (ARIA) with edema/effusion (ARIA-E) and hemorrhage/hemosiderosis (ARIA-H) are side effects unique to this class of drugs that generally do not cause symptoms but can be serious and life-threatening. Carriers of one or two copies of the ApoE4 gene may be at increased risk of developing Alzheimer's disease and experiencing ARIA. Patients should discuss any safety concerns with their physician.