The European Commission (EC) has approved UCB’s Zilbrysq (zilucoplan) as an add-on therapy for certain patients with generalised myasthenia gravis (gMG), a rare autoimmune disease with a global prevalence of 100 to 350 cases per one million people.

The marketing authorisation, which specifically applies to adults who are anti-acetylcholine receptor (AChR) antibody-positive, makes Zilbrysq the first once-daily subcutaneous, targeted component 5 (C5) complement inhibitor for gMG.

Patients with gMG can experience a variety of symptoms, including severe muscular weakness that can result in double vision, drooping eyelids, difficulty swallowing, chewing and talking, and life-threatening weakness of the muscles of respiration.

UCB’s Zilbrysq inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action, the company said, adding that the therapy can be used simultaneously with intravenous immunoglobulin and plasma exchange without the need for supplemental dosing.

The EC’s decision was supported by positive results from the late-stage RAISE study, which demonstrated that Zilbrysq delivered "rapid, consistent, clinically meaningful and statistically significant" improvements in patient- and clinician-reported outcomes at week 12 in a broad population of adult patients with mild-to-severe anti-AChR antibody-positive gMG, UCB said.

The marketing authorisation comes less than two months after Zilbrysq was approved by the US Food and Drug Administration, also for adult gMG patients who are anti-AChR antibody-positive.

Jean-Christophe Tellier, UCB’s chief executive officer, said: “With the EC’s approval of [Zilbrysq], I’m very excited that UCB is taking another important step forward in delivering patient value to the gMG community, giving clinicians an opportunity to address complement activation in gMG with a once-daily, self-administered, subcutaneous treatment option.”

Beyond Zilbrysq, UCB’s neonatal Fc receptor (FcRn) blocker rozanolixizumab was recently recommended by the European Medicines Agency’s (EMA) human medicines committee as an add-on therapy for adults with gMG who are anti-AChR or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive.

If approved, rozanolixizumab will be the first emerging therapy approved in Europe for adults with both anti-AChR and anti-MuSK antibody-positive gMG – the two most common subtypes of gMG.

“Alongside a positive Committee for Medicinal Products for Human Use opinion from EMA for our FcRn blocker rozanolixizumab, and approvals for both [Zilbrysq] and rozanolixizumab in the US and Japan for the treatment of adults with gMG, our unique and differentiated portfolio of medicines reinforces our commitment to redefining treatment expectations for the gMG community,” Tellier said.

https://www.pmlive.com/pharma_news/ec_approves_ucbs_zilbrysq_as_generalised_myasthenia_gravis_add-on_therapy_1504591