CellCept and Myfortic Linked to Pregnancy Loss and Birth Defects

The FDA has issued a warning to healthcare providers and patients that use of the drugs CellCept (mycophenolate mofetil) and Myfortic (mycophenolic acid) during pregnancy is associated with an increased risk of first trimester pregnancy loss and congenital malformations, particularly external ear and facial abnormalities including cleft lip and palate, and anomalies of the distal limbs, heart, esophagus, and kidney. 

CellCept and Myfortic are part of a class of drugs called immunosuppressants and are used to prevent organ rejection in transplant patients. CellCept, manufactured by Roche Laboratories, Inc., is approved to prevent heart, liver and kidney transplant rejection. Myfortic, manufactured by Novartis Pharmaceuticals Corp., is approved to prevent kidney transplant rejection.  Both drugs have also been used off-label to treat immune-mediated conditions such as systemic lupus erythematosus (SLE) and erythema multiforme. Because mycophenolate mofetil (MMF) is converted to mycophenolic acid (MPA), the active ingredient in Myfortic following oral of IV administration, both drugs carry the same risks. 

Based on postmarketing data from the United States National Transplantation Pregnancy Registry which showed 15 miscarriages and 4 birth defects among 33 pregnancies with exposure to MMF, the pregnancy category for CellCept and Myfortic has been changed to Category D (positive evidence of fetal risk) and a "black box" warning of the risk of pregnancy loss and congenital malformations was added to the labels of both drugs last year. 

FDA Ongoing Investigation into Possible Link to PML

The FDA is also currently investigating a potential association between the use of CellCept and Myfortic, and the development of progressive multifocal leukoencephalopathy (PML), a rare, life-threatening disorder affecting the central nervous system, usually occurring in patients with suppressed immune systems.  Many patients who develop PML do not survive.  Those who do may have permanent disability due to irreversible nerve damage. 

Signs and symptoms of PML can include vision changes, loss of coordination, clumsiness, memory loss, difficulty speaking or understanding what others say, and weakness in the legs.