- NMOSD are autoimmune demyelinating diseases targeting the optic nerves, spinal cord, and certain brain areas, often mistaken for MS.
- The Pharmaceutical Benefits Advisory Committee (PBAC)met in November to consider adding ravulizumab (Ultomiris®) to the Pharmaceutical Benefits Scheme (PBS) for adults with NMOSD, which would make this treatment affordable.
- The PBAC has made a groundbreaking recommendation for ravulizumab to be subsidised for treating adults with NMOSD. This is a historic milestone as it’s the first-ever treatment specifically for NMOSD to be recommended for PBS inclusion.
The Pharmaceutical Benefits Advisory Committee (PBAC) has announced its position on subsiding a new treatment for neuromyelitis optica spectrum disorders (NMOSD) after its November meeting.
NMOSD are forms of autoimmune demyelinating diseases that specifically attack and inflame optic nerves, the spinal cord and some other specific locations in the brain. Early in the disease course, NMOSD can easily be mistaken for MS, but it is a different immune-mediated disease with distinct treatment and management approaches.
People with NMOSD have antibodies in their blood that attack a protein called aquaporin-4 (AQP4) in astrocytes (a type of brain cell), damaging optic nerve cells. The complement system, a part of the body’s immune system, facilitates these attacks by destroying cells with a membrane attack complex.
Current treatments for NMOSD involve managing acute attacks and preventing relapses. Specific treatments for NMOSD are not yet on the PBS, and general immunosuppressants used to prevent relapses are prescribed off-label.
In a groundbreaking development, the PBAC has recommended listing ravulizumab (Ultomiris®) on the PBS for adults with NMOSD, marking a revolutionary step forward in NMOSD care.
The PBAC is required to consider several factors when making recommendations, including clinical trial data, community submissions, and pricing information. MS Australia provided support for this submission to the PBAC, which can be viewed here.
How does ravulizumab work and what does the clinical trial show?
Ravulizumab works by blocking the complement system, preventing the destruction of astrocytes.
A recent phase 3 clinical trial showed that ravulizumab significantly reduces relapses in people with NMOSD who are AQP4 antibody-positive compared to those taking a placebo (mock treatment). Additionally, people on ravulizumab experienced less mobility-related disability, with most side effects being mild to moderate. Ravulizumab is administered intravenously every eight weeks in adults, following a loading dose.
What does this mean?
This positive decision is the first step in a process that will now move to the Federal Government for final approval.
MS Australia continues to advocate for systemic changes that will improve the lives of people with MS and MS-related disorders. It remains committed to ensuring that all Australians living with MS and MS-related disorders can benefit from a healthcare system that is inclusive, accessible, and equitable.
