- UCB announced the U.S. Food and Drug Administration granted Breakthrough Therapy Designation for its drug CIMZIA.
- The designation targets the prevention of placenta-mediated adverse pregnancy outcomes in at-risk pregnant women with antiphospholipid syndrome.
- UCB stated the designation is supported by preliminary clinical evidence from the investigator-initiated IMPACT study.
BRUSSELS, Belgium — UCB announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for CIMZIA (certolizumab pegol). The designation is for preventing placenta-mediated adverse pregnancy outcomes (APOs) in at-risk pregnant adult women with antiphospholipid syndrome (APS) who are also positive for lupus anticoagulant (LA) and receiving standard anticoagulant therapy.
The company said in an emailed press release today that these patients were considered at risk due to prior obstetric and blood clotting events. The designation builds on a recent FDA Orphan Drug Designation for CIMZIA in APS.
Addressing Unmet Need
The BTD recognizes CIMZIA’s potential to address a significant unmet medical need. There are currently no FDA-approved therapies for preventing adverse pregnancy outcomes in patients with APS. APS is a rare autoimmune disorder primarily affecting women of childbearing age, associated with an increased risk of blood clots and serious pregnancy complications. These complications include recurrent pregnancy loss, pre-eclampsia, placental insufficiency, fetal growth restriction, preterm birth, and stillbirth.
IMPACT Study Findings
The designation is supported by preliminary clinical evidence from the investigator-initiated IMPACT study. Results from this study demonstrated CIMZIA’s potential to prevent adverse pregnancy outcomes in high-risk women with APS, specifically those with LA positivity and a history of obstetric or thrombotic manifestations of the disease. The findings suggest tumor necrosis factor (TNF)-mediated inflammation contributes to APS-related pregnancy complications.
Breakthrough Therapy Designation
The FDA grants Breakthrough Therapy Designation to medicines for serious or life-threatening conditions. Preliminary clinical evidence must indicate the therapy may offer substantial improvement over available treatments on one or more clinically significant endpoints. This designation aims to facilitate development and expedite the FDA review process for promising new therapies. CIMZIA is not currently approved for use in pregnant women with APS, and its safety and efficacy for this specific use have not been established.