FDA Grants Fast Track Designation to J&J’s Nipocalimab to Reduce Risk of FNAIT in Alloimmunized Pregnant Adults

Rare Daily Staff    

The U.S. Food and Drug Administration granted Fast Track designation for Johnson & Johnson’s nipocalimab to reduce the risk of fetal neonatal alloimmune thrombocytopenia in alloimmunized pregnant adults during their current pregnancy.

The FDA’s Fast Track program is designed to expedite development and review timelines of drugs that demonstrate the potential to treat serious conditions and address unmet medical needs for serious or life-threatening conditions. Fast Track designation supports close communication between the FDA and sponsor with the aim of delivering new therapeutics to patients more quickly.

“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 Katie Abouzahr, vice president, Autoantibody and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson.

Fetal neonatal alloimmune thrombocytopenia (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. If a severe bleed occurs in the brain, termed intracranial hemorrhage (ICH), death, or life-long neurologic effects could occur. ICH occurs in up to 26 percent of untreated pregnancies with FNAIT.

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.

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.

Johnson & Johnson is conducting research and development for nipocalimab, an FcRn blocker, to address the significant unmet need in reducing the risk of FNAIT. Nipocalimab is believed to work by blocking the transfer of immunoglobulin G (IgG) alloantibodies from pregnant individuals to their babies through the placenta while not suppressing the broader immune systems of the pregnant individual or developing fetus. Johnson & Johnson is also proceeding with two phase 3 trials focused on FNAIT. Nipocalimab was granted orphan drug designation by the U.S. FDA for FNAIT in December 2023.

Nipocalimab is also being studied in hemolytic disease of the fetus and newborn (HDFN), another rare alloimmune disease of pregnancy with a similar disease mechanism, often referred to as the red blood cell counterpart to FNAIT. After phase 2 safety and efficacy results from the UNITY trial, Johnson & Johnson is proceeding with phase 3 trials focused on HDFN.

Photo: Katie Abouzahr, vice president, Autoantibody and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson