FDA Fast Tracks Johnson & Johnson’s Nipocalimab for Fetal Neonatal Alloimmune Thrombocytopenia


Johnson & Johnson is moving forward with a pair of Phase III trials of nipocalimab to reduce the risk of fetal neonatal alloimmune thrombocytopenia in alloimmunized pregnant patients.

Image credit: pressmaster | stock.adobe.com

Image credit: pressmaster | stock.adobe.com

The FDA has granted Fast Track designation (FTD) Johnson & Johnson’s nipocalimab to lower the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in alloimmunized pregnant patients.1 The investigational therapy is a high affinity, fully-human, aglycosylated, effectorless, monoclonal antibody that selectively blocks the Fc receptor, which safely decreases autoantibody levels in the mother and blocks maternal autoantibodies from gaining entry to, and potentially harming, the fetus.

“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,” Katie Abouzahr, MD, Johnson & Johnson vice president, Autoantibody and Maternal Fetal Immunology Disease Area Leader, said in a press release. “We are committed to applying our decades of immunology leadership to pioneer innovative approaches to transform treatment for patients and their families affected by FNAIT and other alloantibody-driven diseases of pregnancy.”1

FNAIT is a rare disease in which the immune system of a pregnant individual attacks platelets in the developing fetus, which can have a negative impact on clotting and bleeding, causing a significant risk of severe bleeding complications to the fetus or newborn. There are currently no approved targeted therapies to manage FNAIT, which is not routinely screened for during pregnancy, with diagnoses typically occurring postnatally in firstborn children.

Investigators hypothesize that nipocalimab acts by blocking the transfer of immunoglobulin G alloantibodies from pregnant individuals to the fetus via the placenta without suppressing the broader immune systems of either the mother or developing fetus. Johnson & Johnson is moving forward with a pair of Phase III trials of the drug in the treatment of FNAIT.

In 2019, nipolcalimab was granted FTD in the United States and orphan medicinal product designation by the European Medicines Agency for the treatment of hemolytic disease of the fetus and newborn (HDFN). It was granted Orphan Drug designation in 2020 for this indication.2

Based on Phase II safety and efficacy results from the global, multicenter UNITY (NCT03842189) trial, Johnson & Johnson is subsequently proceeding to Phase III clinical trials for the drug in HDFN. The findings of the trial suggest that nipocalimab helped 54% of alloimmunized patients to achieve a safe life birth at or following 32 weeks of gestation without needing intrauterine transfusion throughout pregnancy.3

The Phase III AZALEA (NCT05912517) trial will evaluate the efficacy of nipocalimab in reducing fetal anemia with live neonates in pregnant individuals with a risk of severe HDFN. Investigators are enrolling individuals aged 18 to 45 years with an estimated gestational age from week 13^0/7 to week 16^6/7 at the time of treatment randomization.

Enrollment criteria includes a history of HDFN, presence of maternal alloantibody to RhD, Rhc, RhE, or RhC antigen with titers above the critical level, and who are healthy based on physical examination, medical history, vital signs, and clinical laboratory tests.3

"Nipocalimab represents a novel approach for the treatment of patients at risk of severe HDFN who need proven, safe, non-surgical solutions to help address the serious health consequences of this condition," Abouzahr said in a prior press release.2


Johnson & Johnson’s nipocalimab granted US FDA Fast Track designation to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in alloimmunized pregnant adults. News release. Johnson and Johnson. March 26, 2024. Accessed March 27, 2024. https://www.jnj.com/media-center/press-releases/johnson-johnsons-nipocalimab-granted-u-s-fda-fast-track-designation-to-reduce-the-risk-of-fetal-neonatal-alloimmune-thrombocytopenia-fnait-in-alloimmunized-pregnant-adults

2. Johnson & Johnson highlights innovation in hemolytic disease of the fetus and newborn (HDFN) at the Society for Maternal-Fetal Medicine’s (SMFM) 2024 Pregnancy Meeting. Janssen. News Release. February 9, 2024. Accessed on March 27, 2024. https://www.janssen.com/johnson-johnson-highlights-innovation-hemolytic-disease-fetus-and-newborn-hdfn-society-maternal

3. A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN) (AZALEA). ClinicalTrials.gov identifier: NCT05912517. Updated February 26, 2024. Accessed March 27, 2024. https://clinicaltrials.gov/study/NCT05912517

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