PBAC resubmissions for Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan) for all Myasthenia Gravis patients

The Australian Myasthenia community have a once in a generation opportunity to support new treatments for generalised Myasthenia Gravis (gMG) which are listed on PBAC’s March 2025 agenda! The Myasthenia Alliance Australia (MAA) encourages Myasthenia patients and your carers or families/friends to consider making a personal submission highlighting your lived experience by 29 January 2025. Why? Because the patient voice matters.

Please also register for the MAA webinar on Wednesday 15 January 2025 so that you can hear directly from expert neurologists and patient advocates. You will learn about the types of treatments on offer, how to make a successful submission to PBAC for medications of your choice and understand why your voice will make a difference. It is ok if you cannot make this date because the webinar will be recorded, but you do need to register. We thank Argenx for supporting this webinar.

The Pharmaceutical Benefits Advisory Committee (PBAC) will assess resubmissions for treatments Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan) for acetylcholine receptor autoantibody positive (AChR+) gMG patients at its meeting in March 2025. Both treatments are already approved by the Therapeutic Goods Administration (TGA) for use and this PBAC meeting further considers the funding and prescribing criteria for gMG patients.

The MAA will make a submission on your behalf as the organisation representing all Myasthenia patients in Australia. Individual submissions add to this by demonstrating the diversity of our experienceswith Myasthenia and the importance of having different treatment options to give us the best care and quality of life. No matter what form of Myasthenia you have, your feedback is valuable. We appreciate this may mean you have to think about some tough or difficult times and experiences, so please allow yourself space to do this or talk to someone about it.

What does the resubmission mean?

Both treatments first went to PBAC for gMG in March and July 2024. They were not approved at the time as more information is required from the pharmaceutical companies sponsoring them. So, they are resubmissions for the March 2025 meeting.

Don’t worry if you made a submission already. Your submission for the previous PBAC meetings is still on the table and will again be considered at this next meeting. You may like to make a submission this time for either or both treatments for a variety of reasons: you may not have been diagnosed earlier this year, or you may have missed making a submission, or you may have some updated information or views you would like to share.

It is important to note that after these meetings in 2024, PBAC has recognised the high clinical need for effective therapies for gMG. This is reinforced by 4 new treatments for gMG being on the March 2025 agenda. More information will follow about the other 2 treatments.

Making a submission to PBAC

Who: Myasthenia Gravis patients (and their carers or families/friends) can make a consumer submission to contribute to the PBAC evaluation of Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan). PBAC is the regulatory body that recommends new medicines for listing on the Pharmaceutical Benefits Scheme (PBS) based on medical conditions, clinical effectiveness, safety, and cost-effectiveness.

What: This is a once in a generation opportunity for new treatments for MG. Ultomiris® (Ravulizumab) and Zilbrysq® (Zilucoplan) are complement C5 inhibitor treatments. Please read more details about these two treatments below.

When: Make a submission by Wednesday, 29 January 2024. We suggest jotting down your thoughts for now, reading the guidance such as this provided by the MAA, and then attending our webinar on 15 January 2025. This still allows 2 weeks to finalise and lodge your submission before the closing date.

How: You can complete the online form here. Please note that you make a submission for each of the treatments separately, not all together. You can make a submission for one or all 4 treatments and we encourage you to think about your preferences across them all.

If you have issues with using the online form, you can send an email directly to commentsPBAC@health.gov.au. If you have difficulties accessing or using technology, please consider asking for help from family, friends or your local librarians.

Why: Because the patient voice matters. The MAA believes this is a once in a generation opportunity to share our voice and lived experience with national decision makers for new treatment options for MG. We want to ensure they are available, accessible, and affordable. This aligns with Priority 2.4 of the National Strategic Action Plan for Rare Diseases: Enable all Australians to have equitable access to the best available health technology.

What should you consider when making a submission?

Your submission to PBAC is guided by 5 questions and can be broken down into two parts:

In the first part, you tell your story about living with MG and the current treatments you take/receive.
In the second part, you focus on the advantages and disadvantages of the specific treatment.

This means you share your experience of living with MG. You do not have to have had or tried using the treatments to make a submission and you do not need to discuss the technical or scientific detail. Talk about your unmet needs with current treatments, the desired benefits of the new treatment, and the outcomes that matter most to you as a patient… if you can imagine any treatment providing you with better management of your symptoms and better quality of life, this is where you can talk about it! Have courage and clearly state your preferences when it comes to your treatment – this could be comparing benefits as well as highlighting any adverse events or side effects that are less acceptable to you than others. You should also explain how the burden of your disease can be reduced.

Ravulizumab**

Ravulizumab is a long-acting complement C5 inhibitor treatment that is being proposed as an add-on to standard therapy (including immunosuppressants like corticosteroids, methotrexate, mycophenolate, and azathioprine) for AChR-positive generalised MG patients. Ravulizumab represents a new pathway in treating MG in a safe and fast-acting manner as demonstrated by its clinical trial. It is also a manageable option for MG patients; generally an 8-weekly treatment cycle of 45-minute infusions.

For more information, please see the Consumer Medicine Information, a TGA-approved document, and the following Australian Government link: https://www.healthdirect.gov.au/medicines/brand/amt,1405341000168107/ultomiris

Zilucoplan**

Zilucoplan is a once daily, self-administered subcutaneous injection of a complement C5 inhibitor treatment. A subcutaneous injection is an injection into the tissue between the skin and the underlying muscle. Zilucoplan is being proposed as an add-on to standard therapy for AChR-positive generalised MG adult patients. Zilucoplan represents a new pathway to treat MG in a fast-acting and manageable manner. The daily self-administered injection can provide flexibility for MG patients by reducing hospital visits and for planning extended travel.

A key update since the July 2024 PBAC meeting is that Zilucoplan has been approved by the TGA, so please see the Consumer Medicine Information, a TGA-approved document.

The resubmission will focus on the treatment of REFRACTORY gMG (those of us who do not respond to current treatments adequately). The MAA strongly encourages this group of MG patients to make a submission for this treatment.

Please also take a look at the previous MAA guidance on both treatments, including questions to think about and tips for writing a submission to PBAC from the Patient Voice Initiative (PVI).

You can also view the public summary documents from the previous PBAC meetings here: https://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/psd/pbac-public-summary-documents-july-2024

We emphasise that this is a once in a generation opportunity! Keep following updates via the MAA website (subscribe here) or your state association. State association members can also access the discussion about treatments from the 2023 MAA conference (please contact your state association if you need mor information about this access). And don’t forget to register for our webinar on Wednesday 15 January 2025 to learn more about the types of new treatments and how to make a valuable submission so that your patient voice is heard.

Kind regards,
Susan White (MAA Chairperson)
Natalie Windle (MAA Secretary & MGNSW President)
Carol Buchanan (MAA Board Member & MGAQ President)

More information and tips about making a PBAC submission can be found through the Rare Voices Australia (RVA) and Patient Voice Initiative (PVI) websites:

https://rarevoices.org.au
https://www.patientvoiceinitiative.org and
https://www.patientvoicehub.org/training/index.cfm?event=page.login

**Please note the MAA and state associations do NOT provide medical advice regarding medications or treatments for individuals. Decisions about whether Ravulizumab or Zilucoplan will be suitable for you as a patient must be discussed with your doctor or health professional. The focus of these submissions to PBAC is to describe our patient perspective of living with MG and where/how/why we feel we have unmet needs, uncertainty, and ongoing burden with current treatment options.