>

Investigational Drug Shows Promise in Treating Rare Fetal Blood Disease

Less Invasive Approach Could Change the Standard

Austin, Texas

Data from a new investigational drug trial show promise for treating a rare blood disease in fetuses and newborn babies. The disease, called non-immune hydrops fetalis (NIHF), is caused by maternal antibodies that attack the fetus's red blood cells. This can lead to severe anemia and swelling of the fetus's body and organs. NIHF is often fatal.

The new drug, called riviparin, is a monoclonal antibody that binds to the maternal antibodies and prevents them from attacking the fetus's red blood cells. In a Phase 2 clinical trial, riviparin was shown to be safe and effective in preventing NIHF in fetuses and newborn babies. The trial results were published in the journal Nature Medicine.

The trial involved 20 pregnant women who were at high risk for having a baby with NIHF. The women were given riviparin once a week during the second and third trimesters of pregnancy. None of the babies in the trial developed NIHF. The most common side effect of riviparin was a rash at the injection site.

The results of the trial are very promising and suggest that riviparin could be a safe and effective treatment for NIHF. If approved by the FDA, riviparin could change the standard of care for this rare and devastating disease.