The European Union’s medicines regulator is reversing course on its decision to block a new Alzheimer’s disease treatment, saying the drug can be offered to certain patients under strict safety protocols.
In late March, the European Medicines Agency’s (EMA) drug evaluators said the risks did not outweigh the benefits for the medicine donanemab, which can slow the progression of early-stage Alzheimer’s but also raises the risk of potentially deadly brain swelling and bleeding.
But in a revised opinion on Friday, the group said donanemab can be offered to a specific subset of Alzheimer’s patients in tightly controlled settings.
Donanemab, which is sold by Eli Lilly as Kisunla, is a monoclonal antibody taken via a once-monthly infusion. It’s already been approved in the United Kingdom, the United States, Japan, and China.
In a clinical trial, Kisunla slowed dementia symptoms by up to 35 per cent over 18 months – but three people died with serious amyloid‑related imaging abnormalities (ARIA), which are a common side effect of the medicine that can cause swelling and bleeding in the brain.
Two of these patients carried a type of gene that raises the risk of Alzheimer’s, and Lilly had suggested that the medicine only be offered to people who do not have the gene.
In March, the EMA committee said that people without the gene were still at risk of fatal complications from ARIA.
On Friday, the group said the drug can be offered to early Alzheimer’s patients who have at most one copy of the gene – but only in a controlled access programme with medical teams that know how to detect and treat ARIA. Patients must also start at a lower dose.
The EMA said that with those measures in place, its advisory group “concluded that Kisunla’s benefits outweigh its risks in noncarriers and people with just one copy” of the gene.
The European Commission has the final say on whether a new drug or vaccine is approved, but it usually adopts the EMA evaluators’ recommendations.
That’s why drugmaker Lilly had asked the EMA group to reconsider its negative opinion on Kisunla. It argued that ARIA is usually temporary and does not cause symptoms in most cases.
Alzheimer’s groups were also disappointed by the initial decision. At the time, Alzheimer Europe said that while patient safety is important, strict prescribing rules and safety monitoring could help ensure dementia patients have access to the new drug while keeping it away from those at higher risk of serious side effects.
Notably, the EMA and the Commission recently greenlit another Alzheimer’s drug, Leqembi, that comes with similar side effects for people with the gene.
In that case, the EMA group had also first refused the drug, but later recommended that it be offered to people with only one or no copies of the gene – making it the first medicine of its kind authorised in the EU.
The Commission is expected to make a final decision on Kisunla in the coming months.
