Price Negotiations Begin for NMOSD Treatment "Uplizna"

The treatment for neuromyelitis optica spectrum disorder (NMOSD), "Uplizna" (inebilizumab), has entered price negotiations three years after its approval. This follows the Drug Reimbursement Evaluation Committee's condition that deemed it cost-effective only if priced below a specific evaluation amount.

NMOSD is a rare autoimmune disease causing unpredictable recurrences of optic neuritis and myelitis, leading to severe disabilities such as blindness and paralysis. Without proper treatment, 60% of patients experience relapse within a year, and 90% within three years.

Uplizna, a humanized monoclonal antibody, targets CD-19, a surface antigen on B cells. By binding to CD19, it depletes B cells responsible for producing anti-aquaporin-4 (AQP4) antibodies, preventing disease recurrence. Approved by the Ministry of Food and Drug Safety (MFDS) in August 2021, Uplizna is indicated for adults with AQP4 antibody-positive NMOSD.

In the "N-MOmentum" clinical trial with 230 NMOSD patients, 89% of those treated with Uplizna remained relapse-free, reducing the risk of recurrence by 77.3% compared to the placebo group. Extended studies showed a relapse-free rate of 87.7% for at least four years.

Following these clinical results, Uplizna passed the Drug Reimbursement Evaluation Committee in July and has now entered price negotiations. The committee's cost-effectiveness assessment suggests it may be listed at a lower price than the company initially anticipated.

Currently, other NMOSD treatments reimbursed in Korea include Enspryng and Soliris, covered as third- and fourth-line treatments, respectively. However, concerns remain that restrictive reimbursement criteria limit the effective use of these treatments. Even if Uplizna is listed for reimbursement, revising these criteria remains a challenge to be addressed.