Empaveli safely manages hard-to-control PNH during pregnancy
Case report: Baby born without complications, shows no signs of medication
Empaveli (pegcetacoplan) was safely administered during a woman’s pregnancy to manage hard-to-control paroxysmal nocturnal hemoglobinuria (PNH), according to a recent study.
Results showed that Empaveli effectively controlled the mother’s PNH, and the baby was born without complications. There were no detectable signs of the medication in cord blood or maternal milk.
The report, “Paroxysmal Nocturnal Hemoglobinuria in Pregnancy Treated With Pegcetacoplan: Case Report and Pharmacokinetic Analysis,” was published in eJHaem. The work was funded by Sobi, which developed Empaveli in collaboration with Apellis Pharmaceuticals.
Empaveli blocks C3 complement protein
PNH is a rare blood disorder in which the complement cascade, a component of the immune system, becomes abnormally activated, leading to the destruction of blood cells and resulting in symptoms such as anemia.
Some PNH treatments work by blocking a complement protein called C5. This is usually effective for blocking blood cell destruction inside blood vessels, known as intravascular hemolysis. But blocking C5 isn’t as effective at stopping blood cell destruction outside of blood vessels, known as extravascular hemolysis, so patients with considerable extravascular hemolysis may experience persistent symptoms despite C5-inhibiting treatments.
Empaveli works by blocking a different complement protein, C3. Inhibiting this protein can help prevent both intravascular and extravascular hemolysis. In clinical trials, Empaveli was shown to ease anemia in most patients whose disease was not adequately controlled with a C5 inhibitor.
The use of any medication during pregnancy requires careful consideration of the potential benefits and risks, as well as the unknowns. For many therapies, there isn’t much data on whether they’re safe to use in pregnancy or not.
In this study, scientists in Canada reported the case of a 24-year-old woman who successfully used Empaveli to help manage her disease during pregnancy.
The woman in the report had been diagnosed with PNH in her late teens. She had originally been started on a C5 inhibitor, but continued to experience persistent anemia requiring regular blood transfusions. She eventually switched to Empaveli, which was effective for managing her anemia and allowed her to avoid transfusions.
Woman gives birth to healthy baby boy
While on Empaveli, the woman became pregnant. The pregnancy was not planned, and since there is no data on whether Empaveli can be safely used in pregnancy, the woman and her doctors decided to switch her back to her previous treatment shortly after learning of the pregnancy. However, she again developed anemia to the extent that she required regular blood infusions throughout the first two trimesters of her pregnancy.
Eventually, after careful consideration of the risks and benefits, the woman and her doctors collaboratively made the choice to switch back to Empaveli around the start of the third trimester. Shortly after switching back, the patient’s blood tests stabilized, and her anemia remained well-controlled through the rest of her pregnancy while she was still on Empaveli.
This report provides the first clinical and [pharmacological] evidence suggesting that third-trimester use of [Empaveli] may be a viable treatment option in select pregnant patients with PNH with suboptimal response to C5 inhibitors.
The woman gave birth by elective cesarean section to a healthy baby boy. Both the birth itself and the postpartum period were uncomplicated for mother and infant, the researchers noted.
“In a patient with hemolytic PNH … conception on [Empaveli] was not associated with adverse pregnancy outcomes, and third-trimester reinitiation resulted in rapid hematological improvement, transfusion independence, and an uncomplicated peripartum course,” the researchers wrote.
The scientists also conducted pharmacological analyses on blood collected from the baby’s umbilical cord and on the mother’s breast milk. Empaveli was not detectable in either, indicating that the therapy did not cross the placenta and was not excreted into milk. The researchers emphasized it’s not possible to draw broad conclusions from this single case, but they noted that this report demonstrates that Empaveli can be used safely during pregnancy.
“This report provides the first clinical and [pharmacological] evidence suggesting that third-trimester use of [Empaveli] may be a viable treatment option in select pregnant patients with PNH with suboptimal response to C5 inhibitors,” the scientists wrote, emphasizing a need for more research efforts to collect data on the use of this therapy during pregnancy.
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