Ultomiris gets expanded indication for neuromyelitis optica

strengthening its competitiveness in the market

Approved for the treatment of adult patients with anti-aquaporin-4 (AQP-4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD)

In the Phase 3 CHAMPION-NMOSD trial, it reduced the risk of recurrence by 98.6%

With an 8-week interval treatment, Ultomiris improves convenience compared to Soliris

Ultomiris (ravulizumab) has strengthened its position in the market for neuromyelitis optica after obtaining approval for expanded indication.

 

Product photo of Ultomiris

On July 11th, the Ministry of Food and Drug Safety (MFDS) granted approval of expanded indication for Ultomiris, a C5 complement inhibitor, for the treatment of adults aged 18 years and above with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP-4) antibody-positive.

 

The approval of NMOSD indication was based on data from the external placebo-controlled, multi-center, open-label phase 3 CHAMPION-NMOSD trial, which evaluated the treatment effect and safety of Ultomiris.

 

The placebo used in the control group was the placebo from Soliris’ phase 3 PREVENT trial for NMOSD, considering that NMOSD is a rare disease and Ultomiris and Soliris are similar treatment types.

 

The data from the 73-week (median treatment period) clinical trial demonstrated that patients who received Ultomiris were recurrence-free and had a 98.7% reduction in the risk of recurrence compared to those who received placebo.

 

Furthermore, the research confirmed a significant improvement in the secondary endpoints, annual recurrence rate (APR) and the Hauser Ambulatory Index (HAI).

 

During the clinical trial, there were no cases with recurrence when treated with Ultomiris, recording 0.000 APR.

 

It was reported that the rate of patients who experienced worsened HAI in Ultomiris was 3.4% (2 out of 58), whereas those who received placebo had a 23.4% HAI (11 out of 47).

 

Additionally, the trial had three severe adverse cases after the start of treatment.

 

Two patients had meningococcal infections but continued treatments after recovery without any side effects.

 

Ho Jin Kim, Professor of the Neurology department at the National Cancer Center, said, “Ultomiris demonstrated no recurrences in NMOSD patients for 73.5 weeks.

 

It is a treatment option with improved convenience of treatment, as it extends the duration interval from 2 weeks to 8 weeks.” Ultomiris is the next-generation C5 complement inhibitor, as it extended its half-life by four times compared to Soliris.

 

Soliris required administration every two weeks, whereas Ultomiris improved the convenience of treatment by extending the interval to 8 weeks.

 

“The interval of treatment not only reduces the number of hospital visits, but also saves the physical strength of patients with difficulties in walking and vision.

 

It also reduces other costs related to hospital visits,” Kim added.

 

“Improvements of convenience alleviate the burden of treatment and help improve patients’ quality of life and treatment compliance,” Kim explained.

 

With the current indication approval, Ultomiris can be used to treat four rare diseases, including ▲paroxysmal nocturnal hemoglobinuria (PNH) ▲atypical hemolytic uremic syndrome (aHUS), and ▲generalized myasthenia gravis (gMG).

 

Chul Woong Kim, Lead for Rare Diseases at AstraZeneca Korea, said, “After obtaining reimbursement approval for Soliris, we are pleased to contribute to the improvement of NMOSD treatment in South Korea with the expanded indication for Ultomiris.” Kim added, “We will strive to enhance treatment options so that more NMOSD patients can receive treatments without fear of recurrence and continue with their daily lives.”