Johnson & Johnson's Milestone FcRn Blocker Nipocalimab (IMAAVY) Approved by FDA for Generalized Myasthenia Gravis at $12,480 per Vial

May 03, 2025 13:56 CST Updated 13:56

Johnson & Johnson

Medical Device R&D and Manufacturer

On April 30 local time, Johnson & Johnson announced that Nipocalimab (IMAAVY) had been approved by the U.S. FDA for marketing to treat generalized myasthenia gravis.（gMG）Patient.

This is the first and only FcRn blocker approved for the treatment of anti-AChR antibody and anti-MuSK antibody positive adult and pediatric gMG patients aged 12 years and above.

Source: Johnson & Johnson official website

Myasthenia Gravis（MG）It is an acquired autoimmune disease, with approximately 700,000 patients worldwide. Among them, about 85% of patients will develop generalized myasthenia gravis.（gMG）, characterized by fluctuating weakness of skeletal muscles, leading to symptoms such as limb weakness, ptosis, diplopia, and difficulties in chewing, swallowing, speech, and breathing.

In patients with MG, the immune system mistakenly attacks proteins at the neuromuscular junction, producing pathogenic IgG antibodies, such as anti-acetylcholine receptor antibodies.（AChR）Antibody, Anti-Muscle Specific Tyrosine Kinase（MuSK）Antibody, Anti-Low Density Lipoprotein-Related Protein 4（LRP4）Antibodies. These antibodies interfere with or block the signal transmission between nerves and muscles, leading to muscle weakness.

Clearing IgG antibodies can effectively control the progression of MG.FcRn plays a key role in extending the serum half-life of IgG.FcRn antibodies can block their binding with IgG, accelerate the catabolism of IgG, lead to a reduction in IgG levels, including the production of pathogenic autoantibodies, thereby treating diseases.

Previously, two FcRn antibodies have been approved globally for the treatment of myasthenia gravis: efgartigimod from Argenx/Zai Lab, and rozanolixizumab from UCB.

Unlike efgartigimod and rozanolixizumab, which also belong to the FcRn antagonists,The recently approved Nicallimab is an FcRn blocker. It can specifically bind to FcRn, block its interaction with IgG antibodies, reduce the recycling and circulation of IgG antibodies, and fundamentally decrease the level of autoantibodies in the blood.

Source: Insight Database by DXY

gMG is the first approved indication for Nicalimab. In terms of dosage form, both intravenous and subcutaneous formulations of Efgartigimod have been approved. As latecomers, Johnson & Johnson and UCB have directly launched subcutaneous injection formulations to enhance the competitiveness of their products. Compared with intravenous infusion, which usually takes about 1 hour,Subcutaneous injection completes drug administration within 30 to 90 seconds, which can greatly improve the convenience of drug delivery and patient compliance.

In terms of indications, nicalumab, like rozelimab, can cover both AChR antibody-positive and MuSK antibody-positive patients. These two types of antibody-positive patients account for over 90% of the total antibody-positive population with gMG, covering a broader population than efgartigimod.(Limited to patients positive for AChR antibodies)。

The FDA approval of Nicalumab was based on the results of the pivotal Phase III clinical study, Vivacity-MG3. The study enrolled a total of 199 patients who were unresponsive to current standard therapies.（SOC）Antibody-positive adult gMG patients with poor response. The primary endpoint is the Myasthenia Gravis Activities of Daily Living score for antibody-positive patients during weeks 22, 23, and 24 of treatment.（MG-ADL）Changes from baseline, secondary endpoints include Quantitative Myasthenia Gravis Score.(QMG ) etc.

The relevant research paper was published inThe Lancet Neurology

Vivacity-MG study data shows:

At weeks 22, 23, and 24 of treatment, patients receiving "Nicalimab"+SOC」, the MG-ADL score improved by 4.70 points, significantly higher than the 3.25-point improvement in the "placebo + SOC" group (P=0.002);
In terms of the secondary endpoint QMG, compared with placebo + SOC, the OMG score improvement was 4.86 points for nicarlimab + SOC (vs 2.05 points in the placebo group), with significant improvements in strength and function across different muscle groups in patients (P<0.001).
The proportion of patients with ≥2-point improvement in MG-ADL scores was 68.8% in the Nicalimab + SOC group, compared to 52.6% in the placebo + SOC group (P=0.021), further indicating that treatment with Nicalimab can reduce the impact of gMG on patients' daily lives.
In terms of safety, nicarlimab was generally well-tolerated, with an overall incidence of adverse events similar to the placebo + SOC group.

David Lee, M.D., Global Head of Immunotherapy at Johnson & Johnson, stated: "Today's FDA approval of IMAAVY represents a significant milestone for more than 240 million patients suffering from autoimmune antibody diseases.It is a historic milestone.」

Nicalimab treatment for gMG also demonstrates long-term efficacy and safety. The ongoing Phase III open-label extension study（OLE）The results showed that gMG patients receiving nicarlimab in combination with standard therapy remained in good condition during a follow-up period of up to 20 months.

At the same time, Johnson & Johnson is also conducting a Phase II/III pediatric study for adolescent gMG patients aged 12-17 who are positive for anti-AChR and anti-MuSK antibodies.（Vibrance-MG）. The study data showed that nicarlimab in combination with SOC therapy achieved its primary endpoint, reducing total serum IgG levels by 69% within 24 weeks, with secondary endpoints MG-ADL and QMGc scores both showing improvement.

Source: Johnson & Johnson official website

The approval of Nicallidumab is significant for Johnson & Johnson. In October 2020, Johnson & Johnson acquired it for $6.5 billion.(Equivalent to approximately RMB 44.8 billion at the average exchange rate at the time)The acquisition of Momenta Pharmaceuticals was primarily aimed at bringing Nicallidumab under its wing, and now it has finally come to fruition. It will become an important catalyst for Johnson & Johnson's next wave of growth in the autoimmune field.

Johnson & Johnson has set the price of Nicarylmab at $12,480 per 1200-milligram vial (approximately RMB 90,746). Johnson & Johnson predicts that the annual sales of Nicarylmab could eventually exceed $5 billion.

Outside the United States, an application for the marketing of nicarlimab for myasthenia gravis has been submitted in Europe, Japan, and China. In China, nicarlimab for gMG has been granted priority review by the CDE, which is expected to accelerate its approval.

Planners: Xinyao, Kenden Yang

References:

[1] Johnson & Johnson Official Website

[2] Insight Database

[3]https://www.biopharmadive.com/news/johnson-johnson-nipocalimab-fda-approval-imaavy-myasthenia-gravis/746709

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