New Study Testing Omaveloxolone in Pregnant Patients, Rozanolixizumab Improves Ocular Symptoms, Efgartigimod to be Studied in Juvenile MG

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio. This week’s episode is centered around the 2025 MDA Clinical & Scientific conference.

Investigators have unveiled the design of a new post-marketing observational study (NCT06628687) assessing maternal, fetal, and infant outcomes following omaveloxolone (Skyclarys; Biogen) exposure during pregnancy and/or lactation. Omaveloxolone, a nuclear factor-like 2 activator, is the only therapy FDA-approved for adults with Friedreich ataxia (FA). This descriptive, phase 4 study is expected to include 10-20 participants, aged at least 16 years, who’ve had exposure to omaveloxolone at any time during pregnancy (from 12 days prior to conception to pregnancy outcome) and/or during lactation (up to 1 year of infant age or weaning, whichever comes first). The study will use prevalence of major congenital malformations, observed up to 10 years, as the primary study outcome, with other secondary outcomes that include minor congenital malformations, neonatal death, gestational diabetes, and others.

In a post-hoc analysis of the previously completed phase 3 MyacarinG study (NCT03971422), results showed that treatment with rozanolixizumab (Rystiggo; UCB), an FDA-approved medication, may enhance ocular symptoms in patients with generalized myasthenia gravis (gMG). These findings were presented as a poster at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas Texas, as well as at the ICNMD, held October 26-29, 2024. Overall, the analysis comprised 200 patients from the study, testing changes of Ocular Muscle Weakness scores on the Myasthenia Gravis Symptoms Patient-Reported Outcomes (MGSPRO) over a 7-day treatment period. Additionally, the study also used Myasthenia Gravis Impairment Index (MGII) ocular subdomain, an 8-item assessment, as an optional outcome over a 14-day period.

At the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19 in Dallas, Texas, a poster presentation highlighted the design of a new study (NCT06392386) testing subcutaneous efgartigimod coformulated with recombinant hyaluranidase (PH20 SC; Vyvgart; Argenx) in juvenile patients with myasthenia gravis (MG), the most common form of pediatric MG. The study, which aims to confirm the age-appropriate dose of efgartigimod PH20 SC, will recruit at least 12 patients, aged 2-17 with a confirmed diagnosis of Myasthenia Gravis Foundation of America class II, III, or IVa. Spanning a total of 14 weeks, the study includes a 2-week screening period, 4-week treatment period, and 8-week follow-up period testing 4 once-weekly injections of efgartigimod PH20 SC. The hope is that results from this study will help address an unmet need and provide additional flexibility for the treatment of pediatric patients with generalized MG.

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