Company Pulls Alzheimer’s Drug Application After EU Rejection
Anavex withdrew its application for approval of its experimental Alzheimer’s drug, after the European Medicines Agency declined to recommend it.
A disease-modifying pill for Alzheimer’s could be a gamechanger. Oral pills would be less expensive, easier to access, and simpler than anti-amyloid drugs, which require intravenous infusions and MRI scans to initiate treatment. But so far, experimental pills have faltered in trials.
Anavex Life Sciences was hoping to buck that trend, but withdrew its bid for approval on March 25. The decision came after it failed late last year to impress the Committee for Medicinal Products for Human Use — the European Medicines Agency’s committee that assesses new drugs.
How well does blarcamesine work?
Blarcamesine is a small molecule drug that turns on a protein called the sigma-1 receptor in brain cells. It triggers a process called autophagy that helps brain cells get rid of brain build-ups of amyloid, tau, and other junk. In the trial, the drug positively altered one blood biomarker of Alzheimer’s and slowed brain shrinkage.
In a 48-week randomized clinical trial completed in 2022, the drug showed mixed results for early Alzheimer’s, slowing cognitive but not functional decline.
More than a third of participants initially given the drug dropped out of the trial — higher than in trials for anti-amyloid drugs Leqembi and Kisunla. The most common side effects of the medication were confusion, dizziness and loss of balance. However, since the drug doesn’t target amyloid directly, it avoids complications like ARIA, brain swelling and small brain bleeds, which are side effects of existing anti-amyloid medications.
Anavex filed their drug application specifically for people with the common version of the gene encoding the sigma-1 receptor protein.
Why did the regulatory committee vote no?
The committee declined to recommend the drug’s approval in December because it failed to meet its main objective: Improving both cognitive and functional outcomes. The committee also cited “methodological issues” as well as safety limitations, stemming from the lack of information on the people who dropped out of the study due to side effects.
The European Medicines Agency pointed to potential quality issues with the drug, writing that it was not possible to rule out the formation of impurities that may cause cancer.
After the feedback, Anavex decided to withdraw the drug application. A press release stated that they are still committed to developing the drug as an Alzheimer’s treatment.
UPDATE: March 26 from earlier version to reflect Anavex Life Sciences’ withdrawal of its bid for approval.
