Johnson & Johnson Seeks FDA Nod For New Myasthenia Gravis Treatment

Johnson & Johnson has submitted a Biologics License Application (BLA) to the U.S. FDA, aiming for the first global approval of nipocalimab as a treatment for generalized myasthenia gravis (gMG). The submission is backed by data from the Phase 3 Vivacity-MG3 trial, which demonstrated that a broad group of antibody-positive participants who received nipocalimab alongside standard care (SOC) showed significantly better outcomes than those who received a placebo with SOC. 

The study's primary endpoint assessed the change in MG-ADL scores from baseline over 24 weeks. Participants included adults positive for anti-AChR, anti-MuSK, and anti-LRP4 antibodies, representing about 95% of the gMG population. Notably, Vivacity-MG3 is the first study to show sustained disease control across these subtypes. The safety and tolerability profile of nipocalimab was consistent with previous studies.

Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Johnson & Johnson Innovative Medicine, said in a statememt, “We are encouraged by the potential of nipocalimab to provide sustained disease control for people living with generalized myasthenia gravis, a chronic, life-long disease.   

He further added, “The filing for approval of nipocalimab represents an important step forward as Johnson & Johnson continues to push the boundaries of research to develop innovative solutions to treat autoantibody-driven diseases, building on decades of expertise in neuroscience and immunology. We look forward to working with the FDA in their review of the data supporting the submission.”

Nipocalimab is a drug designed to block FcRn, with the goal of achieving long-term disease management. This is measured by improvements in MG-ADL scores when it is used alongside standard of care (SOC) treatment compared to a placebo plus SOC over six months of consistent bi-weekly dosing. This represents the most extended period of evaluated safety and effectiveness for an FcRn blocker in generalized myasthenia gravis (gMG) to date.

Earlier this year, at the American Academy of Neurology Annual Meeting, Johnson & Johnson showcased research highlighting the unique molecular features of nipocalimab. The drug exhibits a strong affinity and specificity for the immunoglobulin G (IgG) binding site of FcRn, setting it apart from other FcRn blockers. These attributes, along with the study's dosing schedule, are believed to reduce IgG levels, including IgG autoantibodies in conditions such as gMG and other autoimmune diseases driven by autoantibodies.

Myasthenia gravis (MG) is an autoimmune disorder where the body's own antibodies mistakenly attack proteins at the neuromuscular junction. This interference disrupts the communication between nerves and muscles, leading to impaired or prevented muscle contraction. The immune system produces antibodies against various proteins, such as anti-acetylcholine receptor (AChR), anti-muscle-specific tyrosine kinase (MuSK), or anti-low-density lipoprotein-related protein 4 (LRP4), which block or disrupt the normal signaling process. MG affects approximately 700,000 people globally, impacting individuals of all genders, ages, and ethnicities, though it most commonly affects young women and older men. About 50% of those diagnosed with MG are women, and around 20% of these women are of childbearing age.

Initially, MG symptoms often appear in the eyes, but in more than 85% of cases, the condition progresses to generalized MG (gMG). This form is marked by fluctuating muscle weakness that can lead to issues such as limb weakness, drooping eyelids, double vision, and difficulties with chewing, swallowing, speaking, and breathing. In the U.S., approximately 100,000 individuals are affected by gMG. While current treatments can manage the condition, there is a need for new therapies for those who do not respond adequately to or cannot tolerate existing options.