An investigative therapy for hemolytic disease of the fetus and newborn (HDFN) has just received the FDA’s Rare Pediatric Disease and Orphan Drug Designations. This therapy is called nipocalimab and its developer, Momenta Pharmaceuticals, believes it holds a lot of promise.

About HDFN

HDFN is a rare condition which can occur during pregnancy. It occurs when the placenta transfers alloantiboties from the mother to the child. These antibodies attack the blood cells in the fetus.

HDFN ranges in severity. Depending on its level of severity, it can cause early birth, fetal anemia, neonatal morbidity, fetal loss, or neonatal loss. For most women, the condition is more severe in subsequent pregnancies. The most severe cases require an intrauterine blood transfusion. This procedure in and of itself carries risk. It is an invasive procedure that has a 20% rate of mortality for the fetus.

There is clearly a need for a safer treatment option which is not invasive and more effective.

Nipocalimab  

Nipocalimab is an anti-FcRnmonoclonal antibody. FcRn is what allows the mom’s IgG to be transferred from her placenta to the child. By targeting FcRn, this transfer can be stopped. Additionally, it lowers the amount of antibodies available to make the transfer in the first place.

This therapy has an insignificant release to the fetus, minimizing the child’s exposure to the drug.

As of now, it is the only therapy being investigated for this disease.

Clinical Trials

There are three ongoing clinical trials investigating nipocalimab.

UNITY Trial

The UNITY Trial is a Phase 2 trial investigating nipocalimab in HDFN. It is currently enrolling patients for five trial sites in the U.S. as well as some in Europe.

The CLARITY trial is also currently enrolling HDFN patients.

ENERGY Trial

The ENERGY trial is a Phase 2/3 investigation of nipocalimab in warm autoimmune hemolytic anemia (wAIHA). The goal is to begin enrolling patients by the last quarter of 2020.

Nipocalimab has already received Orphan Drug Designation and Fast Track Designation for wAIHA.

VIVACITY-MG Trial

The VIVACITY-MG trial is an ongoing study investigating whether nipocalimab is effective for generalized myasthenia gravis (gMG). 8 week data was positive showing no serious AE’s. The drug was found to improve daily living for the patients. Full data from this trial is expected by the last quarter of this year.

You can read more about Nipocalimab and its investigations here.