NMOSD patients call for Ultomiris’s reimbursement

“Even a single relapse must be prevented"

Ultomiris remains out of reach due to non-reimbursement despite demonstrating a relapse-free period of 73.5 weeks

Secured long-term relapse-free outcomes and treatment convenience…focus on domestic reimbursement

Calls grow for policy measures to improve treatment access

With the emergence of an innovative treatment option for neuromyelitis optica spectrum disorder (NMOSD), a disease in which even one relapse can cause a dramatic decline in quality of life, voices are growing for improved access.

 

Specifically, voices are rising on the need to improve access to Ultomiris (ravulizumab), which has demonstrated a relapse-free period of 73.5 weeks in a clinical trial, as a paradigm-shifting therapy to overcome the limitations of existing treatments.

 

Need for treatment approaches focused on “relapse prevention” highlighted in NMOSD NMOSD is a central nervous system autoimmune disorder that causes nerve damage, primarily presenting with optic neuritis and myelitis.

 

More than 90% of patients experience relapses.

 

Neuromyelitis optica spectrum disorder (NMOSD) can lead to cumulative nerve damage with repeated relapses, potentially resulting in lower body paralysis or blindness.

Repeated relapses lead to cumulative nerve damage, resulting in symptoms such as ocular pain, vision loss, spinal or limb pain, muscle weakness, gait disturbance, and sensory-motor impairment of the lower body, which may progress to paraplegia or blindness.

 

NMOSD occurs most frequently in women in their 30s and 40s, an age group typically active in the workforce, leading to social isolation caused by employment discontinuation, reduced interpersonal interactions, and limited activity.

 

This has negative repercussions not only on the individual but also on families and society as a whole.

 

For example, patient A first experienced symptoms around the age of 16, but it took 5 years to receive an accurate NMOSD diagnosis.

 

By then, despite devoting herself to treatment, the aftereffects of frequent relapses left her with significant daily limitations.

 

She is currently being treated with MabThera (rituximab), but side effects make even basic tasks such as eating and walking difficult without family assistance.

 

Long-term immunosuppressive therapy has also weakened her immunity, making her prone to colds, pneumonia, and enteritis, and at times she has faced emergencies such as breathing difficulties and shock.

 

In NMOSD treatment, the key is to block relapses at an early stage.

 

Experts in Korea and abroad emphasize that because even a single relapse can cause irreversible damage, treatment approaches aimed at preventing relapse itself are critical.

 

Ultomiris demonstrates “relapse-free” effect in NMOSD Against this backdrop, the next-generation C5 complement inhibitor Ultomiris has gained attention as an innovative therapy, confirming relapse-free efficacy in a Phase III trial with NMOSD patients.

 

By improving its half-life, the drug overcomes limitations of previous therapies, and among 58 patients in the Ultomiris group, the relapse rate during the trial period was 0%, suggesting a paradigm shift in NMOSD treatment.

 

Pic of Ultomiris

In the Phase III CHAMPION-NMOSD study, Ultomiris showed no relapses during a median treatment period of 73.5 weeks and reduced relapse risk by 98.6% compared to placebo.

 

Dosing intervals are also noteworthy: Ultomiris can be administered every 8 weeks, significantly reducing treatment burden compared to Soliris, which requires dosing every 2 weeks.

 

Professor Ho-Jin Kim from the Department of Neurology at the National Cancer Center explained, “The extended dosing interval not only reduces hospital visits, it also lowers physical strain and additional costs associated with travel for patients with impaired mobility and vision.

 

Such improvements in treatment convenience can enhance patients’ quality of life and adherence to therapy.” However, the fact that Ultomiris is yet to be reimbursed in Korea limits actual treatment access.

 

Accordingly, both clinical experts and the Korean NMOSD Patient Association are increasingly calling for guaranteed access to proven therapies.

 

Patient A said, “I live with daily anxiety, never knowing when a relapse will strike.

 

I know there is a therapy that prevents relapses, but cannot try it because it is not reimbursed.” Meanwhile, to promote proper understanding and social awareness of NMOSD, a “Stay ZERO” concert will be held on September 25 at the National Assembly Member Office Building.

 

The event aims to amplify the voices of patients longing for a “relapse-free life,” raise awareness of the importance of relapse-prevention treatment, and emphasize the need for improved treatment accessibility.