Warning Added for Progressive Multifocal Leukoencephalopathy to CellCept and Myfortic

Roche and Novartis have added warnings to their organ transplant medications, CellCept and Myfortic, indicating an association with Progressive Multifocal Leukoencephalopathy, a rare and potentially fatal neurological condition.

The label changes were announced over two months after the FDA issued an early communication in April 2008 to announce that they were investigating reports of users who developed the life-threatening disease

According to letters Novartis and Roche sent to healthcare providers dated June 2008, at least 17 cases of PML have been identified among users of CellCept, and at least seven of the cases had fatal outcomes. No confirmed cases have been associated with Myfortic, but the drugs could carry the same risk in humans, since the active pharmaceutical ingredient in CellCept (MMF, or mycophenolate mofetil) converts to the active ingredient in Myfortic (MPA, or metobolite mycophenolic acid).

Progressive Multifocal Leukoencephalopathy (PML), which is also known as Progressive Multifocal Leukoencephalitis, is a rare viral disease that causes progressive damage or inflammation at multiple locations in the brain. Symptoms could include weakness, paralysis, vision loss, impaired speech or cognitive deterioration. Although the disease does slow or stop in some cases with improvements in the immune system, there is no known cure and the disease is often fatal.

CellCept and Myfortic are immunosuppressant medications often prescribed after organ transplants to help prevent the body from rejecting the organ. It is also often prescribed “off-label” to treat lupus, which is not an approved use.

The drugs have also been associated with increased risk of fetal injury, such as cleft palate, cleft lip and ear deformities, when taking during pregnancy. The manufacturers added a blackbox warning about the Myfortic and CellCept birth defect risks in November 2007, and the FDA re-posted an alert on their website in May 2008, since many doctors remain unaware of the danger of fetal injury.