Commentary
Article
Empowering Patients: Exploring Self-Administration of Rozanolixizumab for Myasthenia Gravis
Author(s):
Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at Wake Forest School of Medicine, discussed a new, innovative trial assessing the therapeutic potential of self-administered subcutaneous rozanolixizumab as a treatment for myasthenia gravis.
Rachana K. Gandhi Mehta, MBBS
In June 2023, the FDA made a landmark decision, approving UCB’s rozanolixizumab-noli as the first treatment for both anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive generalized myasthenia gravis (gMG), the most common subtypes of gMG. The basis for the approval was data from the phase 3 MycarinG study (NCT03971422), in which rozanolixizumab-treated patients showed significant reductions in the primary end point of Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores (P <.001) over a 43-day period.1,2
Slightly more than a year later, at the 2024 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 15-18, in Savannah, Georgia, investigators presented an outline of a new phase 3 study assessing a self-administered version of rozanolixizumab using a syringe and manual push method. Known as MG0020, this open-label, crossover study randomly assigned patients to once-weekly rozanolixizumab for 18 consecutive weeks consisting of a 6-week self-administration training period, followed by two 6-week self-administration periods. After training, patients are randomly assigned 1:1 to the syringe driver or manual push self-administration method, subsequently crossing over to the alternative method.3
To better understand more about this innovative study, NeurologyLive® sat down with lead investigator Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at the Wake Forest School of Medicine. Mehta, an expert in neuromuscular disorders, highlighted the growing interest in patient-centered therapies that enhance independence and quality of life. In the discussion, Mehta covered the trial’s design, patient preferences, and potential benefits of self-administration, such as reduced reliance on healthcare providers and greater flexibility in daily life.
NeurologyLive: How did this study come about?
Rachana K. Gandhi Mehta, MBBS: As you know, rozanolixizumab is a neonatal Fc receptor blocker that has already demonstrated efficacy in reducing disease burden with an acceptable side effect profile in clinical trials. It is now FDA-approved for acetylcholine receptor and MuSK-positive myasthenia gravis patients. Currently, it’s administered subcutaneously by a healthcare provider, with infusions given weekly for six weeks during each cycle. This means patients must either visit an infusion center or rely on infusion nurses for treatment.
There has been growing interest among patients in having more patient-centered therapy options—not only to manage the clinical aspects of myasthenia gravis but also to improve their quality of life. If patients could self-administer rozanolixizumab, they’d gain more independence, allowing them to do infusions at home without relying on nurses or traveling to an infusion center.
What advantages does this syringe driver hold?
The syringe driver method was actually used in the MycarinG study, though it was handled by nurses. One great aspect of the syringe driver is its ability to deliver a precise dose and control the rate of infusion. It’s portable and easy to use with proper training. Patients could keep the pump in their pocket and go about their day—working on a computer, reading a book, or watching TV—all while receiving their medication.
Is there any learning curve to using this syringe driver?
Yes, but patients in the trial received six weeks of training—three weeks for the syringe driver and three weeks for the manual push method. The patients we enrolled learned both methods very easily, which was encouraging.
How was the trial conducted?
This was a Phase III open-label, randomized study exploring the self-administration of rozanolixizumab in patients with generalized myasthenia gravis. In the study, patients underwent six weeks of training—learning both the syringe driver method and the manual push method. Following this comprehensive training, participants were randomized 1:1 to either the syringe driver or the manual push method for six weeks. Afterward, they crossed over to try the alternate method, completing weekly rozanolixizumab infusions for a total of 18 consecutive weeks.
The study assessed several endpoints. The primary endpoint was the success of self-administration, defined as patients’ ability to select the correct site, administer the medication subcutaneously, and deliver the correct dose. Secondary endpoints included adverse reactions, injection site reactions, MG-ADL scores, IgG levels, medication errors, and patient preferences—both between self-administration and healthcare provider-administered infusions, as well as between the two self-administration methods.
What does the timeline of the trial look like?
The study has already concluded, and researchers are currently analyzing the data. We should have the results available very soon.
Were there any notable inclusion/exclusion criteria for this study?
Yes, it followed standard trial protocols. It enrolled both seropositive and seronegative generalized myasthenia gravis patients, as long as they were willing to try the self-administration methods.
How does this study reflect the state of myasthenia gravis treatment today?
In recent years, we’ve seen the approval of many new targeted treatments for myasthenia gravis, such as complement inhibitors and FcRn receptor blockers. This reflects a shift toward therapies that target the immune system more precisely compared to traditional immunotherapies.
If self-administration options become available for these treatments, it could significantly enhance patients’ quality of life. It would allow them greater flexibility and independence, reducing the need for frequent clinic visits and reliance on healthcare providers. This study highlights how we are not only improving the clinical outcomes of myasthenia gravis but also addressing the broader goal of patient-centered care.
