The FDA has granted Fast Track Designation (FTD) to nipocalimab (M281; Johnson & Johnson) for reducing fetal neonatal alloimmune thrombocytopenia (FNAIT) risk in pregnant alloimmunized adults, according to Johnson & Johnson.
Takeaways
- Nipocalimab, developed by Johnson & Johnson, has been granted Fast Track Designation by the FDA for reducing the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in pregnant alloimmunized adults.
- FNAIT is an autoimmune condition in pregnancy where antibodies attack fetal platelet antigens, leading to low platelet counts in newborns and severe bleeding complications, including intracranial hemorrhage (ICH), which can result in lifelong disability or death.
- ICH occurs in up to 26% of untreated pregnancies with FNAIT, highlighting the urgent need for targeted therapies.
- Nipocalimab is a monoclonal antibody designed to decrease immunoglobulin G antibody levels by blocking FcRn, offering potential treatment for FNAIT and other rare autoantibody diseases.
- Johnson & Johnson is also exploring the efficacy of nipocalimab in treating hemolytic disease of the fetus and newborn after promising results from a phase 2 trial.
FNAIT is an autoimmune condition in pregnant patients where the immune system creates antibodies that attack fetal or newborn platelet antigens. This causes low platelet counts in offspring and may lead to severe bleeding complications in the gastrointestinal tract, lungs, or eyes that can cause lifelong disability or death.
Severe bleeding in the brain may lead to lifelong adverse neurological outcomes, termed intracranial hemorrhage (ICH), or death. ICH has been reported in up to 26% of untreated pregnancies with FNAIT, and there are currently no approved targeted therapies for FNAIT. FNAIT diagnosis often occurs postnatally because of a lack of screening during pregnancy.
Nipocalimab is a monoclonal antibody designed to decrease immunoglobulin G antibody levels by blocking FcRn. Currently, it is the only FcRn blocker being studied for use against rare autoantibody diseases, maternal fetal diseases mediated by alloantibodies, and prevalent rheumatology.
The FTD will allow the development and review timeline of nipocalimab to accelerate. It is granted to drugs with the potential to treat severe conditions and provide unmet vital medical needs. The designation allows for close communication between the FDA and the drug manufacturer, allowing patients to receive treatment sooner.
Johnson & Johnson is also evaluating the efficacy of nipocalimab for treating hemolytic disease of the fetus and newborn (HDFN), a disease characterized by incompatibility of certain maternal-fetal red blood cell types. This leads to fetal red blood cells being attacked by alloantibodies that cross the placenta during pregnancy.
Adverse outcomes of HDFN include fetal anemia and newborn hyperbilirubinemia and anemia which can be life-threatening. After a phase 2 UNITY trial showed safety and efficacy from nipocalimab against HDFN, Johnson & Johnson is preparing for a phase 3 trial.
Reference
Johnson & Johnson's nipocalimab granted U.S. FDA Fast Track designation to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in alloimmunized pregnant adults. PR Newswire. March 26, 2024. Accessed March 26, 2024. https://www.prnewswire.com/news-releases/johnson--johnsons-nipocalimab-granted-us-fda-fast-track-designation-to-reduce-the-risk-of-fetal-neonatal-alloimmune-thrombocytopenia-fnait-in-alloimmunized-pregnant-adults-302099499.html
