AstraZeneca has moved a step closer to launching a new rare disease treatment in Europe.
The FTSE 100 drugmaker said Klygefa, developed by its Alexion rare disease arm, had won a positive recommendation from the European regulator's key advisory committee.
The Committee for Medicinal Products for Human Use, which advises the European Medicines Agency, backed the drug as an add-on therapy for adults with generalised myasthenia gravis.
The condition, known as gMG, is a rare autoimmune disorder that causes severe muscle weakness and can, at its worst, lead to breathing failure.
If the recommendation is rubber-stamped, Klygefa would become the first treatment of its kind cleared in the EU for the roughly 66,000 eligible patients across the region's five largest markets.
Its selling point is convenience, with a once-weekly injection that patients can administer themselves at home.
Klygefa is a so-called nanobody, a compact antibody fragment tuned to dampen the immune system's misfiring attack on muscle.
That marks a shift from the intravenous infusions long associated with the drug class.
The green light rests on the late-stage PREVAIL trial, which met its main goal by improving patients' ability to carry out everyday tasks compared with a placebo.
Crucially for sufferers, the benefit showed up within a week and held for the full six-month study.
The drug was generally well tolerated, in line with earlier medicines in the same family.
Klygefa builds on Soliris and Ultomiris, the two blockbuster treatments that established Alexion as a pioneer in the field before AstraZeneca bought the business.
It is already approved in Japan, while regulators in the United States and China are still weighing submissions.
For AstraZeneca, the nod adds another string to a rare disease unit that has become an increasingly important growth engine.
A final decision from the European Commission is expected in the coming months.
