Alexion's Soliris approved in new optic nerve disease use in Europe

Alexion’s rare disease drug Soliris has been approved in a new use in Europe, to tackle a disorder of the optic nerves for which it recently got FDA approval.

The European Commission granted a marketing authorisation for Soliris (eculizumab) for adults with neuromyelitis optica spectrum disorder (NMOSD), in adults with an AQP4 antibody with relapsing disease.

This follows approval in the US in the new indication at the end of June.

NMOSD has unpredictable relapses causing potentially irreversible consequences, such as blindness, and inability to walk.

The disease is caused by an overactive complement system, which Soliris helps to inhibit, and prolonging time to relapse.

Although only approved in a handful of rare diseases, the complement-inhibiting drug generated $981 million for Alexion in Q2 alone, up from $898 million in the previous year’s Q2.

Although the disease will likely only affect a few thousand patients in Europe, at the prices Alexion charges this could generate significant revenues.

First approved by the FDA in 2007 for paroxysmal nocturnal haemoglobinuria, Soliris is threatened by the possibility of biosimilar competition as its US patent expires in March 2021 and it goes off patent in Europe in May next year.

Amgen is developing a biosimilar that could undercut Soliris on price if approved and marketed, and Alexion is hoping to maximise Soliris’ revenues in its last years of its patent life, as well as developing a longer-lasting alternative, Ultomiris.

European approval is based on comprehensive results from the phase 3 randomised, double-blind placebo controlled PREVENT trial, where 96 NMOSD patients received Soliris and 47 patients in a comparator arm received placebo.

The study met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse.

At 48 weeks, 98% of patients treated with Soliris were relapse free compared to 63% of patients receiving placebo.

Of the patients treated solely with Soliris, without receiving other immunosuppressive therapies, all were relapse free at 48 weeks compared to 61% in the placebo group. Sustained effects were observed through 144 weeks of treatment.

The safety profile of the drug was consistent with that seen in other clinical studies and real-world use in its three approved indications.