FDA Grants UCB’s Cimzia Breakthrough Designation for High-Risk APS Pregnancies

Rare Daily Staff

The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to UCB’s Cimzia, an anti-inflammatory drug, for preventing serious placenta-related pregnancy complications in a subset of pregnant women with antiphospholipid syndrome, or APS.

FDA Breakthrough Therapy Designation is reserved for medicines intended for serious or life-threatening conditions when preliminary clinical evidence suggests they may offer a substantial improvement over available treatment options on a clinically meaningful outcome. The status provides a company with more intensive FDA guidance and a greater level of agency attention during development.

The FDA decision was supported by preliminary findings from the investigator-sponsored IMPACT study, which evaluated Cimzia in pregnant women with high-risk APS. UCB said the study suggested the drug could help prevent adverse outcomes linked to placental dysfunction. There are no FDA-approved medicines specifically indicated to prevent adverse pregnancy outcomes in women with APS.

APS is a rare autoimmune disorder in which the immune system produces antibodies, known as antiphospholipid antibodies, that can make blood more likely to clot. The condition can cause clots in veins or arteries and disproportionately affects women of childbearing age. During pregnancy, APS can interfere with the placenta, the organ that supplies a developing fetus with oxygen and nutrients. That can raise the risk of recurrent miscarriage, preeclampsia, fetal growth restriction, placental insufficiency, premature delivery and stillbirth.

Pregnant patients with APS are often managed with low-dose aspirin and heparin, an anticoagulant. But some patients—particularly those with lupus anticoagulant and a history of obstetric or thrombotic complications—remain at high risk despite those measures.

Cimzia is a biologic medicine administered by injection. It blocks tumor necrosis factor, or TNF, an immune-system signaling protein that helps drive inflammation. UCB and the IMPACT investigators point to growing evidence that TNF-mediated inflammation may contribute to APS-related pregnancy complications. The company previously received FDA Orphan Drug Designation for Cimzia for the same use.

In the United States, Cimzia is already approved for several immune-mediated diseases, including Crohn’s disease, rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, non-radiographic axial spondyloarthritis, plaque psoriasis and polyarticular juvenile idiopathic arthritis. It is not approved to treat APS or to prevent APS-related pregnancy complications.

Like other TNF blockers, Cimzia carries important safety warnings, including an increased risk of serious infections such as tuberculosis, sepsis and invasive fungal infections. Its U.S. prescribing information also warns of malignancy risk, hepatitis B reactivation, neurologic reactions, blood-related abnormalities and other potential adverse effects. Whether its benefits outweigh those risks for pregnant women with APS remains to be established through further development and regulatory review.