FDA grants fast track designation to Johnson & Johnson’s nipocalimab for rare disease

The US Food and Drug Administration (FDA) has granted fast track designation to Johnson & Johnson’s nipocalimab to reduce the risk of FNAIT, a rare but life-threatening immune disorder, in alloimmunized pregnant adults.

Foetal neonatal alloimmune thrombocytopenia (FNAIT) is a rare and severe condition that occurs when the immune system of a pregnant woman mistakenly attacks platelets in a developing foetus.

This immune response can lead to impaired clotting ability and bleeding, posing a significant risk to the foetus or newborn.

FDA has granted fast track designation for Johnson & Johnson’s nipocalimab, an investigational, high-affinity, fully human, aglycosylated, effectorless, monoclonal antibody (mAb), to reduce the risk of FNAIT in alloimmunizeda pregnant adults during a current pregnancy.

According to a company press release, 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.

“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 foetus or newborn,” said Katie Abouzahr, vice president, autoantibody and maternal foetal immunology disease area leader at Johnson & Johnson.

“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.”

Johnson & Johnson, Abouzahr said, 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 foetus.

The pharmaceutical giant is also conducting two Phase 3 trials focused on FNAIT. Nipocalimab was granted orphan drug designation by the US FDA for FNAIT in December 2023.

Nipocalimab is additionally being studied in haemolytic disease of the foetus and newborn (HDFN), another alloimmune disease of pregnancy with a similar disease mechanism, often referred to as the red blood cell counterpart to FNAIT.