Soliris now approved in China to treat adults with NMOSD

Therapy proven to reduce risk of relapse in AQP4-positive patients

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by Steve Bryson, PhD |

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Soliris (eculizumab) has been approved in China to treat adults with neuromyelitis optica spectrum disorder, known as NMO or NMOSD, who test positive for antibodies against the aquaporin-4 (AQP4) protein.

The therapy — which works to reduce the risk of relapse and ease inflammation among patients — was approved in the Asian nation by the National Medical Products Administration, a Chinese regulatory agency. It will be marketed by its developer, Alexion Pharmaceuticals, now Alexion, AstraZeneca Rare Disease.

“We are proud to bring our first-in-class C5 inhibitor Soliris to the NMOSD community in China, reflecting our commitment to transforming the lives of people living with rare neurological diseases and expanding access to our medicines around the world,” Marc Dunoyer, Alexion’s CEO, said in a company press release.

“Patients with NMOSD and their families should not have to live in fear of the next relapse and potential complications,” Dunoyer said.

Soliris already had been approved for the same patient population in the U.S., Canada, and Japan, as well as in the European Union. It’s also approved in many countries to treat paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and myasthenia gravis, among other diseases.

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Clinical Efficacy of Soliris Supported by Pharmacological Analysis

How Soliris works

In NMO, the immune system mistakenly targets healthy parts of the nervous system. These abnormal immune attacks are typically associated with the presence of self-reactive antibodies targeting AQP4. This protein is found on the surface of cells that support neurons, or nerve cells.

The binding of antibodies to AQP4 causes a chain reaction that results in damaging inflammation. This primarily affects the spinal cord and optic nerves, which transmit signals between the eyes and the brain. As such, common NMO symptoms include painful burning or tingling, involuntary muscle contractions, muscle weakness, and vision problems.

Most people with NMO experience unpredictable relapses, meaning a worsening of symptoms or the onset of new ones. Such relapses can lead to permanent disability.

The chain reaction also forms a group of complement proteins that induce cell death. This formation depends on the splitting of the C5 protein. Soliris is designed to bind to C5, preventing the split.

Through this process, the therapy aims to ease inflammation, lessen symptoms, and reduce relapses. It is administered every two weeks directly into the bloodstream.

Nearly all patients on Soliris remain relapse-free after 3 years

The approval by the Chinese regulators was based on data from the Phase 3 PREVENT clinical trial (NCT01892345). That trial enrolled 143 NMO adults with anti-AQP4 antibodies across 18 countries.

The results showed that 98% of patients treated with Soliris were relapse-free after 48 weeks, compared with 63% of those who received a placebo. This represents a relative risk reduction of 94.2%.

Data collected after nearly three years showed that 96% of Soliris-treated participants remained relapse-free, compared with 45% of those on a placebo.

These benefits were sustained for more than 3.5 years, according to data from PREVENT and its open-label extension study (NCT02003144).

Soliris’ safety profile remained consistent throughout the trial and the extension study. Common side effects included infections and inflammation of the nose and throat, headaches, and nausea.

[Soliris’] approval marks a fundamental shift in the care of NMOSD in China.

“The Phase [3] PREVENT trial established the safety and efficacy of C5 inhibition in reducing the frequency of relapses that can lead to severe and long-term disability for people living with NMOSD,” said Xu Yan, MD, PhD, chief physician of the department of neurology at the Peking Union Medical College Hospital, in China.

“With nearly all patients in the trial achieving relapse-free status at 48 weeks, [Soliris’] approval marks a fundamental shift in the care of NMOSD in China,” Yan added.