Alexion Receives Positive Opinion from EU’s CHMP for Soliris to Treat Subgroup of gMG Patients

Alexion Pharmaceuticals has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion to extend the approved indication of Soliris (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) to patients who are anti-acetylcholine receptor (AChR) antibody-positive, a rare subgroup of gMG patients.

A chronic, progressive autoimmune disease, MG typically begins with weakness of the eye muscles, sometimes progressing into a more generalized form (gMG) that could affect speech, swallowing, vision, breathing, and more.

Most symptoms can be managed, but about a tenth of gMG patients do not respond to conventional therapy and continue to suffer muscle degradation regardless of treatment, in which case they’re called refractory.

In MG patients who are AChR antibody-positive, the body’s own immune system turns against the patient’s body itself and produces antibodies targeting AChR, a receptor located on muscle cells in the neuromuscular junction that nerve cells use to communicate with the muscles.

Soliris is approved in the U.S., EU, and Japan as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) to prevent the continuous destruction of red blood cells (hemolysis) that characterize the disease.

Soliris is also approved as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated blood clots in small vessels.

Soliris has been granted orphan drug status for the treatment of MG by regulatory agencies in the U.S., the European Union, and in Japan.

“Despite existing treatment options for gMG, patients with refractory gMG continue to suffer from severe symptoms and disease complications that significantly impact their daily lives,” John Orloff, MD, executive vice president and head of research and development at Alexion, said in a press release.

“The positive CHMP opinion is a critical milestone in bringing Soliris to patients with refractory gMG who are anti-AChR antibody-positive and for whom physicians currently have no approved therapy,” he said.

The positive opinion was based on comprehensive clinical data from the Phase 3 REGAIN trial (NCT01997229), as well as from its long-term extension study (NCT02301624).

The final decision will be made by the European Commission and is anticipated in the third quarter of 2017. If approved, Soliris will be the European Union’s (EU) first treatment available for this subgroup of patients, as well as the first and only complement inhibitor approved for gMG.

In related news, Alexion’s supplemental biologics license application (sBLA) to the U.S. Food and Drug Administration (FDA) was accepted for review, as was its supplemental new drug application in Japan, to include Soliris as a treatment for these patients.