The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nipocalimab for the treatment of pregnant women at high risk of severe hemolytic disease of the fetus and newborn (HDFN), Johnson & Johnson announced.
Breakthrough Therapy designation is meant to speed up the process of drug development and regulatory review. It is granted to drugs that show potential to treat serious or life-threatening conditions more effectively and/or safely than currently available medications.
“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,” 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.”
Read more about HDFN therapies
This decision is based on clinical evidence from the phase 2 open-label UNITY clinical trial. In this trial, pregnant women at high risk for HDFN who were treated with nipocalimab achieved a live birth at or after the gestational age of 32 weeks, without the need for an intrauterine blood transfusion during the pregnancy.
Moreover, the novel drug was linked with low rates of severe or serious adverse events. Its safety was overall aligned with pregnancy, HDFN and gestational age at birth.
At present, nipocalimab is the only therapy in clinical development for the preventive treatment of HDFN. A phase 2 pivotal AZALEA is currently enrolling pregnant women who are at risk for severe HDFN or who have a history of severe HDFN in previous pregnancies.
The FDA granted the experimental drug a Fast Track designation in July 2019 and an Orphan Drug designation in June 2020. Nipocalimab also received an orphan medicinal product designation by the European Medicines Agency in October 2019 for HDFN prevention.
