FDA Approves Nipocalimab for Generalized Myasthenia Gravis
The US Food and Drug Administration (FDA) has approved nipocalimab (Imaavy) injection for the treatment of adults and children aged 12 and older with generalized myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibodies, as announced by Johnson & Johnson.
“We consistently hear from individuals living with myasthenia gravis who are hopeful for new treatment options that may help bring greater stability, independence and predictability to their lives,” said Samantha Masterson, President and CEO, Myasthenia Gravis Foundation of America. “Today’s announcement provides another option which could help address the constant uncertainty and heavy physical and mental toll that MG symptom relapse presents to patients and their families.”
gMG is a chronic, debilitating autoantibody disease for which significant unmet patient need exists for additional efficacious therapies with demonstrated safety profiles that offer sustained disease control. Anti-AChR and anti-MuSK antibody positive individuals comprise ≥90% of the total antibody-positive gMG population.4 IMAAVY is an immunoselective therapy designed to substantially reduce immunoglobulin G (IgG), including harmful IgG autoantibodies, without additional detectable effects on other adaptive and innate immune functions.
The approval is supported by data from the pivotal, ongoing Vivacity-MG3 study – the longest primary endpoint of a registrational trial of any FcRn blocker in adults with living with gMG. Highlights of the study include:
• IMAAVY plus standard of care (SOC) provided superior disease control throughout 24 weeks when compared to placebo plus SOC, as measured by improvement in the MG ADL score. This translates into patients regaining essential daily functions, such as chewing, swallowing, speaking and breathing.
• Participants on IMAAVY plus SOC maintained improvements out to 20 months of follow-up in the ongoing open-label extension (OLE) study in gMG.
• IMAAVY demonstrated a rapid and sustained reduction in autoantibody levels by up to 75% from the first dose and throughout a 24-week period of monitoring.
“The clinical results we’ve seen with IMAAVY represent a significant milestone in the treatment of gMG,” said Dr. Nicholas J. Silvestri, M.D., Professor of Neurology at University of Buffalo. “Patients experienced substantial symptom relief and lasting disease control that translated into better daily function and did not fade over 24 weeks in the pivotal Vivacity-MG3 study. Having a treatment that delivers this level of durable symptom stability is a meaningful step forward for managing a complex and unpredictable disease like gMG, and to have it in both AChR+ and MuSK+ adults and pediatric patients 12 years and older brings an additional FcRn treatment to a broader range of patients.”
Results from the ongoing Vibrance Phase 2/3 pediatric study in anti-AChR and anti-MuSK antibody positive adolescents aged 12-17 years showed that IMAAVY plus SOC met its primary endpoint with a 69% reduction in total serum IgG over 24 weeks, and secondary endpoints of improvements in MG-ADL and QMGc scales.
IMAAVY has demonstrated a consistent safety profile across both Vivacity-MG3 and the ongoing Vibrance-MG studies, with comparable tolerability in adult and pediatric populations.
“Today’s FDA approval of IMAAVY marks a historic milestone for the more than 240 million patients suffering with autoantibody diseases, many with few or no approved targeted treatments,” said David Lee, M.D., Ph.D., Global Immunology Therapeutic Area Head, Johnson & Johnson Innovative Medicine. “This approval is the result of years of scientific commitment, collaboration and determination for our nipocalimab program, and we’re proud to bring this new treatment option to patients living with anti-AChR or anti-MuSK antibody positive gMG.”
Johnson & Johnson is committed to supporting affordable access to all its treatments, including offering a patient support program called IMAAVY withMe in the United States. With this program, commercially insured patients who are prescribed IMAAVY may be eligible to receive their first treatment in as quickly as one week and may pay as little as $0 per infusion.
Health authority submissions seeking approval for nipocalimab in the treatment of gMG are currently under review with numerous regulatory authorities worldwide.
About Generalized Myasthenia Gravis (gMG)
Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] ), which target proteins at the neuromuscular junction and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction. The disease impacts an estimated 700,000 people worldwide. The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently starts in young women and older men. Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential. Approximately 10 to 15% of new cases of MG are diagnosed in pediatric patients 12-17 years of age. Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the U.S. diagnosed in girls.
Initial disease manifestations are usually eye-related but approximately 85 percent of MG patients experience additional advancements to the disease manifestations-referred to as generalized myasthenia gravis (gMG). This is characterized by severe muscle weakness and difficulties in speech and swallowing. Approximately 100,000 individuals in the U.S. are living with gMG. Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.
