The MGFA community played a key role in the development of a new myasthenia gravis treatment, approved by the FDA in December. Vyvgart (efgartigimod alfa-fcab), produced by argenx, is the first and only FDA-approved neonatal Fc receptor (FcRn) blocker for adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
A Partnership Formed
MGFA first partnered with argenx in 2018, when the company was launching clinical trials for Vyvgart. The company reached out to Nancy Law, then-executive director of the MGFA and an MG patient.
“Nancy was the first MG patient we ever spoke to,” said Rebecca McLeod, argenx’s U.S. general manager.
From their lab benches, argenx scientists had a limited view into the real-life effects of MG or its treatments on patients. To deliver a drug that would truly meet patient needs, they needed to know more about the people being affected.
“What we hoped for was a partnership between us and this patient community that’s been underserved over the years,” said McLeod.
Nancy Law was a huge influence on how the company interviewed patients and created the ultimate individualized dosing design of the clinical trial.
“An MG trial is always difficult because it’s a rare disease,” said Dr. Deb Gelinas, executive director of medical affairs at argenx. “MGFA was really wonderful in sharing information about the trial. Through the MGFA network and conference, we were able to reach health care providers who could speak with patients about joining. MGFA has been a tremendous partner.”
At Nancy’s suggestion, argenx undertook a patient listening tour in the United States. They learned that patients wanted a dose that was specific to their needs and symptom development. These conversations informed how the trial was designed and the drug ultimately delivered.
“We had a lot of options on how to design the trial,” McLeod said. “Initially we were treating with four infusions over four weeks. We saw that some patients had this ongoing duration of benefits. So, we went to Nancy and team and asked how the patient community would like to be treated – individual or standardized? They liked this idea of not taking the medication if they didn’t need it.”
During the trial, patients were offered a flexible dosage depending on how they responded to the drug. Assessment of the trial data indicated that a flexible, individualized dosage offered clinically significant results while also providing a better patient experience.
Novel Drug
Vyvgart reduced harmful AChR antibody that causes symptoms in approximately 68% of myasthenia patients who are AChR antibody positive in the Phase 3 ADAPT trial. That’s sufficient to show real benefits to patients with fewer side effects than other immunosuppressant drugs. (For more on how the treatment works, check out this recent MGFA webinar and see prescribing information: https://argenx.com/product/vyvgart-prescribing-information.pdf
“Vyvgart has an established safety and efficacy profile, and can be dosed in a way that is individualized to the patient,” said Gelinas.
James F. Howard, Jr., a neurology professor at The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the trial, agrees that the FDA approval of Vyvgart represents an important new advance for myasthenia patients and their families.
“People living with gMG have been in need of new treatment options that are targeted to the underlying pathogenesis of the disease and supported by clinical data,” he said. “This therapy has the potential to reduce the disease burden of gMG and transform the way we treat this disease.”
Recognition
Argenx was recognized at the MGFA Patient Conference in February 2022 as MGFA Partner of the Year for their work bringing this novel treatment option to market.
“We were super honored to get this recognition from MGFA – we think that how they show up and serve the community is just outstanding,” said McLeod. “They played such a pivotal role in this.”
MGFA and argenx will continue to work together to support clinical data collection and assessment to best meet the needs of patients. This includes a patient symptom tracker designed to collect real-world evidence that can be used to assess how patients experience myasthenia.
Medical professionals and argenx representatives will be at the MGFA International Conference on Myasthenia Gravis and Related Disorders in May 2022 to talk more about Vyvgart as well as other opportunities to be involved in MG research.