Details
The U.S. Food and Drug Administration (FDA) initiated this study in the Sentinel System to evaluate the use and exposure to mycophenolate during pregnancy. FDA was interested in obtaining real world data about mycophenolate to assist in regulatory decision-making toward modifying the mycophenolate Risk Evaluation and Mitigation Strategy (REMS). This REMS was designed to ensure the benefits of the drug outweigh the risk of embryo-fetal toxicity associated with the use of mycophenolate during pregnancy. This study focused on mycophenolate exposure in females aged 10 to 54 years by disease indication and use during pregnancy.
Results indicated a low overall proportion of pregnancy episodes (604/7,355,770 = <0.01% in the 11-year study period) with exposure to mycophenolate during pregnancy. In both patient groups [1) those with a prior organ transplant (using the drug to prevent organ rejection), and 2) those with an off-label use of the drug (i.e. for lupus, rheumatoid arthritis, scleroderma, vasculitis, or myasthenia gravis)], the use of mycophenolate was lower during pregnancy compared to matched nonpregnant cohorts (approximately 14% vs. 40% in group 1, and 6% vs. 35% in group 2).
The Sentinel study results evaluating the use of mycophenolate during pregnancy, particularly in patients with the indication for which the drug is approved, provided important real-world evidence that helped to inform FDA’s determination that a REMS is no longer necessary for mycophenolate products.