FDA grants breakthrough therapy designation for J&J’s nipocalimab as HDFN treatment

Johnson & Johnson (J&J) has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for nipocalimab for the treatment of alloimmunised pregnant individuals at high risk of severe haemolytic disease of the foetus and newborn (HDFN).
The drug is the only therapy currently in development for this indication, which is a rare condition that can occur when the blood types of a pregnant individual and the foetus are incompatible, leading to potentially life-threatening anaemia in the foetus or infant.
This designation follows data from the proof-of-concept phase 2 open-label UNITY clinical trial, which met its primary endpoint of the majority of pregnant patients receiving the drug achieving a live birth at or after a gestational age of 32 weeks without needing an intrauterine transfusion (IUT) throughout their pregnancy.
Severe or serious adverse events were generally low and remained consistent with events associated with pregnancy, HDFN and gestational age at birth.
Katie Abouzahr MD, vice president, autoantibody and maternal foetal immunology disease area leader at J&J, commented: “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. We are committed to addressing the substantial unmet need in this devastating disease.”
The company’s press release explains that ‘Nipocalimab was granted Fast Track designation in July 2019, orphan drug status in June 2020 by the FDA, and orphan medicinal product designation by the European Medicines Agency in October 2019 for the HDFN indication’.