NMOSD Overview
NMOSD are autoimmune demyelinating diseases that target the optic nerves, spinal cord, and certain brain areas, often mistaken for multiple sclerosis (MS). These diseases involve antibodies attacking the aquaporin-4 (AQP4) protein in astrocytes, leading to damage of optic nerve cells. The complement system, part of the body’s immune system, plays a crucial role in these attacks.
Current Treatment Landscape
Current treatments for NMOSD focus on managing acute attacks and preventing relapses. However, specific treatments for NMOSD are not yet on the PBS, and general immunosuppressants used for relapse prevention are prescribed off-label.
Ravulizumab's Mechanism and Clinical Trial Results
Ravulizumab works by blocking the complement system, thereby preventing the destruction of astrocytes. A recent phase 3 clinical trial demonstrated that ravulizumab significantly reduces relapses in AQP4 antibody-positive NMOSD patients compared to placebo. Patients on ravulizumab also experienced less mobility-related disability, with most side effects being mild to moderate. The treatment is administered intravenously every eight weeks in adults, following a loading dose.
PBAC Recommendation and Next Steps
The PBAC's recommendation to list ravulizumab on the PBS for adults with NMOSD is a groundbreaking development. This recommendation is based on clinical trial data, community submissions, and pricing information. The next step involves the Federal Government's final approval for PBS inclusion.
Advocacy and Support
MS Australia has supported the submission for ravulizumab's PBS inclusion, advocating for systemic changes to improve the lives of people with MS and MS-related disorders. The organization is committed to ensuring that all Australians living with these conditions can benefit from an inclusive, accessible, and equitable healthcare system.
