AstraZeneca's (LSE:AZN) rare disease unit Alexion has secured a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for Klygefa (gefurulimab), an add-on treatment for adults with generalised myasthenia gravis (gMG) who test positive for anti-acetylcholine receptor antibodies.
If approved, Klygefa would become the first and only dual-binding nanobody C5 inhibitor available for this patient group in the EU, offering once-weekly subcutaneous self-administration.
gMG is a rare autoimmune disorder causing severe muscle weakness, with an estimated 82,500 people diagnosed across Germany, France, the UK, Italy and Spain, of whom 66,000 are antibody-positive.
The CHMP opinion rests on the Phase III PREVAIL trial, which enrolled 260 patients across 20 countries and met its primary endpoint, showing a 1.6-point treatment difference on the Myasthenia Gravis Activities of Daily Living scale at week 26 versus placebo (p<0.0001).
Improvement was evident within a week and sustained through the study period, with a safety profile consistent with earlier C5 inhibitors eculizumab and ravulizumab.
"With this positive CHMP opinion patients may soon have the option of a novel treatment option that could help them spend less time thinking about their care and more time living their lives," said Tobias Ruck, an investigator on the trial and director of neurology at Ruhr-University Bochum's Bergmannsheil hospital.
Klygefa is already approved in Japan and other markets, with regulatory submissions under review in the US and China.
News Intelligence what this means for the company
AstraZeneca's Alexion has secured a positive CHMP opinion for Klygefa (gefurulimab) in generalised myasthenia gravis, positioning it as a potential first-in-class dual-binding nanobody C5 inhibitor in the EU. The recommendation rests on Phase III PREVAIL data showing a 1.6-point treatment difference on the Myasthenia Gravis Activities of Daily Living scale at week 26 versus placebo (p<0.0001), with improvement evident within a week and sustained through the study period; formal EU approval is now likely, and the drug is already approved in Japan with submissions under review in the US and China.
This is a regulatory de-risking event for a rare disease asset within Alexion's portfolio. The CHMP opinion removes a material approval uncertainty in a defined patient population (66,000 antibody-positive gMG patients across five major EU markets) and opens a new revenue stream in a therapeutic area where AstraZeneca has demonstrated clinical and regulatory competence with C5 inhibitors.
Insights assembled by AI. Editor-reviewed and grounded in tickstock’s coverage and proprietary knowledge graph.
Content is for informational purposes only, not financial advice.
