FDA Grants Fast Track Designation to Nipocalimab for Fetal Neonatal Alloimmune Thrombocytopenia


In December 2023, nipocalimab (Johnson & Johnson) was granted orphan drug designation for fetal neonatal alloimmune thrombocytopenia.

The FDA has granted fast track designation to nipocalimab (Johnson & Johnson) to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in pregnant adults who are alloimmunized, according to a press release from the company. The drug is the only investigational therapy reported to be in clinical development for this indication.1

Pharmacists checking inventory at hospital pharmacy | Image Credit: Jacob Lund - stock.adobe.com

Image Credit: Jacob Lund - stock.adobe.com

“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, vice president of Autoantibody and Maternal Fetal Immunology Disease Area Leader at Johnson & Johnson, said in the 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

Nipocalimab is an FcRn blocker that works by blocking the transfer of immunoglobulin G alloantibodies from pregnant individuals to the infant through the placenta, according to the press release. Further, the drug does not suppress the broader immune systems of either the individual or the developing fetus.1

Due to the phase 2 safety and efficacy results demonstrated in the UNITY (NCT03842189) trial, the company will proceed with phase 3 clinical trials focused on hemolytic disease of the fetus and newborn (HDFN). The UNITY trial was a phase 2 global, multicenter study that evaluated nipocalimab as a once weekly intravenous infusion for individuals with HDFN. The results showed that nipocalimab helped 54% of alloimmunized women achieve a safe life birth at or after 32 weeks’ gestation without the need for intrauterine transfusion throughout pregnancy.1,2

About The AZALEA Trial

Trial Name: A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN)

ClinicalTrials.gov ID: NCT05912517

Sponsor: Janssen Research & Development LLC

Completion Date (Estimated): July 2027

The AZALEA (NCT05912517) trial is currently recruiting and will assess the efficacy of nipocalimab compared the placebo in decreasing fetal anemia with live neonates in individuals who are pregnant and at risk for severe HDFN, according to the clinical trial information. Individuals aged 18 to 45 years will be included with an estimated gestational age from week 13^0/7 to Week 16^6/7 at the time of treatment randomization. Individuals will also be included if they have a history of HDFN, have a presence of maternal alloantibody to RhD, Rhc, RhE, or RhC antigen with titers above the critical level, and are otherwise healthy based on physical examination, medical history, vital signs, 12-leadECG, and clinical laboratory tests.3

Individuals will be excluded if there is fetal anemia prior to randomization, if they are pregnant with multiple gestation, have uncontrolled hypertension, a history of myocardial infarction, unstable ischemic heart disease, or stroke, and other criteria that investigators have marked for exclusion, according to the clinical trial information.3

In December 2023, nipocalimab was granted orphan drug designation for FNAIT by the FDA. Further, it was also granted breakthrough designation for the treatment of alloimmunized pregnant individuals who have a high risk of early-onset severe HDFN, according to an article in Pharmacy Times.1,2

  1. 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 26, 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. Hunter E. FDA Grants Breakthrough Therapy Designation to Nipocalimab for the Treatment of Rare Disease in Pregnancy. Pharmacy Times. February 9, 2023. Accessed March 26, 2024. https://www.pharmacytimes.com/view/fda-grants-breakthrough-therapy-designation-to-nipocalimab-for-the-treatment-of-rare-disease-in-pregnancy
  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 28, 2024. Accessed March 26, 2024. https://clinicaltrials.gov/study/NCT05912517
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