AstraZeneca's Klygefa receives positive EU approval recommendation

Alexion, AstraZeneca Rare Disease's Klygefa (gefurulimab) has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), recommending its approval in the European Union as an add-on therapy for generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive.

The CHMP based its opinion on results from the Phase III PREVAIL trial, which were published in JAMA Neurology. The trial showed Klygefa met its primary endpoint, demonstrating a statistically significant improvement from baseline in Myasthenia Gravis Activities of Daily Living total score at week 26 compared to placebo (treatment difference: -1.6 [95% CI: -2.4, -0.8], p<0.0001). Improvement was observed as early as week one and was sustained through week 26.

If approved, the company claims Klygefa would be the first dual-binding nanobody C5 inhibitor for this patient population. The treatment is administered once weekly via subcutaneous self-injection using an autoinjector.

Approximately 82,500 people are diagnosed with gMG across five European countries — Germany, France, the UK, Italy, and Spain — with an estimated 66,000 being AChR-positive, according to the press release. gMG is a rare autoimmune disorder characterized by reduced muscle function and severe muscle weakness.

Klygefa was described as generally well-tolerated, with a safety profile consistent with prior trials involving C5 inhibitors eculizumab and ravulizumab in gMG.

The drug is already approved in Japan and other countries for certain adults with gMG. Regulatory submissions based on PREVAIL results are currently under review in the United States, China, and additional countries.

AstraZeneca (NASDAQ: AZN) is the parent company of Alexion, AstraZeneca Rare Disease.

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