EMA Committee Recommended Klygefa for EU Approval
The drug targets adult generalised myasthenia gravis as an add-on to existing standard therapies.
Updated on Sept. 21, 2026 in Alzheimer’s
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The Committee for Medicinal Products for Human Use has issued a positive opinion recommending Klygefa for approval in the European Union. This treatment is intended for adults with generalised myasthenia gravis who test positive for anti-acetylcholine receptor antibodies.
Why it matters
The recommendation aims to provide a new therapeutic option for patients struggling with disease severity and daily function. Klygefa acts as a dual-binding nanobody C5 inhibitor to help manage symptoms by preventing the immune system from over-responding.
The PREVAIL trial, involving 260 patients across 20 countries, showed a statistically significant improvement in MG-ADL scores at week 26 with p<0.0001. Approximately 66,000 patients in five European countries are currently anti-acetylcholine receptor antibody-positive.
The players
Committee for Medicinal Products for Human Use
This body is responsible for evaluating medicinal products for human use within the European Union.
Alexion
This biopharmaceutical company is based in Boston and specializes in developing therapies for rare diseases.
The details
Patients receive a weight-based loading dose on Day 1 followed by weekly maintenance doses to block the C5 protein. This clinical approach is designed to mitigate the overactive immune response characteristic of generalised myasthenia gravis.
Timeline
Patients receive an initial weight-based loading dose on Day 1.
Maintenance dosing begins for patients on Day 8.
Clinically meaningful improvements were observed in patients by Week 1.
The PREVAIL trial primary endpoint was measured at Week 26.
Trial results were shared during the 2025 Annual Meeting.
The Big Picture
The drug intervenes in the terminal complement cascade to stabilize immune function, mirroring the mechanism of other targeted C5 inhibitors. This therapy advances the application of C5 inhibition technology within the broader field of chronic neurological diseases.
Patients diagnosed with generalised myasthenia gravis may soon have access to this new dual-binding nanobody treatment as an add-on to their standard care. While the recommendation is a step toward availability, potential users should monitor clinical guidance for final authorization and specific dosing protocols.
The takeaway
Klygefa represents a potential shift toward more targeted nanobody-based therapies for rare autoimmune conditions. Patients should consult with their healthcare providers regarding how emerging C5 inhibition treatments might fit into their ongoing management plans.
Further reading
Learn more about the latest innovations in neurodegenerative care in the Alzheimer’s section.
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