New Treatment Raises Hopes for Rare Pediatric Disease

A potential treatment breakthrough offers hopeful news to parents of infants with a rare blood disorder.

The FDA has granted rare pediatric disease status and orphan drug status to a new drug for hemolytic disease of the fetus and newborn, or HDFN. The disease occurs when the blood types of a mother and her developing or newborn baby are incompatible. If the mother’s blood type is negative but the baby’s is positive, the mother’s immune system may attack the baby’s “foreign” red blood cells. 

In the most severe cases, anemia, jaundice, congestive heart failure, or even death in the womb may occur. HDFN, which affects 3,000 to 8,000 pregnancies per year, typically becomes more severe with mothers’ subsequent pregnancies. 

A formal designation for rare pediatric disease underscores the value of effective new treatments for the deadly disease. Currently, treatment for severe cases is intrauterine blood transfusions, an invasive procedure that can cause significant complications. In up to 20% of cases, the infant does not survive the procedure.

The new treatment is injected into the bloodstream, allowing patients to receive much-needed care without risking complications from the blood transfusions. The drug candidate has shown positive results thus far. Over an 8-week study, the drug exhibited no serious adverse events and was well tolerated by patients.

By granting pediatric rare disease status, the FDA reflects the “high priority” the agency places on treatments for pediatric patients. Before the FDA launched a pediatrics program, only about 20% of drugs administered to infants and children were actually approved for children. The drug’s designation decision also offers a ray of hope for families facing the rare and potentially devastating disease.

The FDA is fast tracking nipocalimab, with clinical trials expected to begin enrollment this fall.  

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