JOHNSON & JOHNSON FNAIT is a rare and severe condition that occurs when the immune system of a pregnant person mistakenly attacks platelets in a developing fetus. This immune response can lead to impaired clotting ability and bleeding, posing a significant risk to the fetus or newborn.1 Nipocalimab, an investigational monoclonal antibody targeting FcRn, is the only investigational therapy currently reported to be in clinical development to address the needs of alloimmunized pregnant individuals at risk of FNAIT.
The
'Receiving Fast Track designation for nipocalimab in FNAIT underscores the urgency to address the unmet need for safe, effective, and targeted treatments to prevent FNAIT, a condition that could carry severe health consequences and even be fatal for the fetus or newborn,' said
Nipocalimab is additionally being studied in hemolytic disease of the fetus and newborn (HDFN), another alloimmune disease of pregnancy with a similar disease mechanism, often referred to as the red blood cell counterpart to FNAIT.3 After Phase 2 safety and efficacy results from the UNITY trial,
About Fetal Neonatal Alloimmune Thrombocytopenia (FNAIT)
Fetal neonatal alloimmune thrombocytopenia (FNAIT) is a rare and potentially life-threatening alloimmune condition in which a pregnant person's immune system develops antibodies against fetal or newborn platelet antigens, leading to thrombocytopenia (low platelet counts in the fetus or newborn).1
FNAIT can result in severe bleeding complications for a fetus or newborn and is characterized by organ bleeding in the gastrointestinal tract, lungs, or eyes, and may result in lifelong disability or death.1 If a severe bleed occurs in the brain, termed intracranial hemorrhage (ICH), death or life-long neurologic effects could occur.1 ICH occurs in up to 26 percent of untreated pregnancies with FNAIT.5
There are no approved targeted therapies for FNAIT management. FNAIT is not routinely screened for during pregnancy and firstborn children with FNAIT are often only diagnosed postnatally.1
About HDFN
Hemolytic disease of the fetus and newborn (HDFN) is a rare disease (and in its severe form, ultra rare) that arises in pregnancies with maternal-fetal incompatibility in certain red blood cell types.6 Alloantibodies produced by the maternal immune system against fetal red blood cells cross the placenta during pregnancy and attack fetal red blood cells causing fetal anemia or persist after birth in the neonate to cause neonatal hyperbilirubinemia and anemia.2 The symptoms of HDFN can range from mild jaundice, to neurotoxic hyperbilirubinemia in the newborn, to life-threatening fetal anemia requiring invasive intervention.7 The potential for in utero onset at an increasingly earlier GA with increasing risk of severe outcomes may occur with each incompatible pregnancy due to pregnancy-related alloimmunization.8 Currently there are no non-surgical interventions approved for pregnancies at high risk for severe HDFN.3 Pregnancies affected by severe HDFN may necessitate repeated intrauterine transfusions (IUTs), which are invasive, technically complex surgical procedures performed by specialists at specialized medical centers, and these procedures are associated with an increased rate of fetal mortality and premature birth.9,10,11 The most difficult to treat cases of HDFN are early onset severe HDFN (EOS-HDFN) that develops at 24 weeks gestational age (GA) and results in significant fetal/neonatal morbidity and mortality. According to the
About Nipocalimab
Nipocalimab is an investigational, high-affinity, fully human, aglycosylated, effectorless, monoclonal antibody that aims to selectively block FcRn to reduce levels of circulating immunoglobulin G (IgG) antibodies, including autoantibodies and alloantibodies that underlie multiple conditions.13 Nipocalimab is the only FcRn blocker being studied across three key segments in the autoantibody space: Rare Autoantibody diseases (e.g., generalized myasthenia gravis in adults and children, chronic inflammatory demyelinating polyneuropathy, warm autoimmune hemolytic anemia, and idiopathic inflammatory myopathies); Maternal Fetal diseases mediated by maternal alloantibodies (e.g., hemolytic disease of the fetus and newborn and fetal and neonatal alloimmune thrombocytopenia) and Prevalent Rheumatology.14,15,16,17,18,19,20,21,22 Blockade of FcRn has the potential to reduce overall IgG including pathogenic alloantibody levels while preserving immune function without causing broad immunosuppression. Blockade of IgG binding to FcRn in the placenta is also believed to prevent transplacental transfer of maternal alloantibodies to the fetus.8,23
About
At
Cautions Concerning Forward-Looking Statements
This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of
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