SPRING HOUSE, PA — Johnson & Johnson recently announced that its investigational drug, nipocalimab, has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA). The designation is for the treatment of alloimmunized pregnant individuals at high risk of severe hemolytic disease of the fetus and newborn (HDFN), a serious and rare condition.
Hemolytic disease of the fetus and newborn (HDFN) is a condition in which a mother’s antibodies attack her fetus’s red blood cells, causing anemia and other serious complications. It can occur when the mother and fetus have incompatible blood types, leading to the production of antibodies that target the fetus’s blood cells.
Currently, there are no non-surgical interventions approved in the U.S. for pregnancies at high risk of early-onset severe (EOS) HDFN. In cases of severe HDFN, pregnancies may require repeated intrauterine transfusions (IUTs). Furthermore, nipocalimab stands as the sole therapy under clinical development for this particular indication.
“Nipocalimab represents a novel approach for the treatment of patients at risk of severe HDFN who need proven, safe, non-surgical solutions,” said Katie Abouzahr, M.D., Vice President, Autoantibody and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson. “We are committed to addressing the substantial unmet need in this devastating disease.”
The BTD was supported by data from the proof-of-concept Phase 2 open-label UNITY clinical trial. The trial met its primary endpoint, with most pregnant patients who received nipocalimab achieving a live birth at or after the gestational age of 32 weeks, without the need for intrauterine transfusion (IUT) throughout their pregnancy. Severe or serious adverse events were generally low and consistent with events associated with pregnancy, HDFN, and gestational age at birth.
Nipocalimab previously received Fast Track designation by the FDA in July 2019 and orphan drug status in June 2020. The European Medicines Agency also granted it orphan medicinal product designation in October 2019 for HDFN indication.
The FDA’s BTD is intended to expedite the development and regulatory review of an investigational medicine that is designed to treat a serious or life-threatening condition. The designation is granted based on preliminary clinical evidence that the drug may substantially improve at least one clinically significant endpoint over available therapies.
The AZALEA Phase 3 pivotal trial, which is currently enrolling pregnant individuals at risk for severe HDFN with a history of severe HDFN in prior pregnancies, will further investigate nipocalimab’s potential in treating this rare condition.
Industry watchers expect that nipocalimab, if approved, could be a game-changing treatment option for individuals with severe HDFN. The healthcare industry is closely following the progress of the AZALEA trial and eagerly awaiting results.
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