Roche and Novartis are two drug companies that make the same medication used to help organ transplant patients better accept a donor organ. CellCept and MyFortic are both the generic drug mycophenolic acid, and they act as immunosuppressant drugs, giving patients a better chance of not rejecting an organ. This medication has been in use in the U.S. since the 1990s, but since then it has been shown to cause serious side effects in some patients.

These CellCept side effects include severe infections and more rare conditions like progressive multifocal leukoencephalopathy and pure red cell aplasia. Most troubling for patients is that mycophenolic acid may contribute to the risk of birth defects when used by women while pregnant. Because of these devastating potential side effects, the drug makers have come under fire for not adequately warning the public.

What is Mycophenolic Acid?

Mycophenolic acid, also sometimes referred to as mycophenolate, is a compound that researchers first found over 100 years ago. At the time it seemed to have potential as an antibiotic or as a drug to treat fungal infections, although research stalled and these uses were never pursued much further. It wasn’t until the 1970s that mycophenolic acid was rediscovered and tested clinically as an immunosuppressant, or a drug that can suppress or limit the action of the immune system.

Roche makes and sells CellCept, while Novartis makes MyFortic. They are both mycophenolic acid, although in slightly different forms. The U.S. Food and Drug Administration (FDA) approved the use of mycophenolic acid in 1995. The agency approved it for use in people receiving a donor kidney, liver, or heart. Other drugs are supposed to be used in conjunction with CellCept or MyFortic, to help ensure that the patient’s body will not reject the new organ. Off-label, doctors may prescribe these drugs for people with autoimmune disorders like multiple sclerosis or lupus.

Both CellCept and MyFortic can be given to patients as a tablet or an oral suspension, a liquid medication. CellCept is also available as an intravenous injection. Children over five years old may be given these medications six months after a transplant surgery. Allergies are a concern with both medications. Some people may have a serious reaction to mycophenolic acid and cannot use it. In addition to the potential for allergic reactions, this medication can cause some serious and troubling side effects in some patients.

Birth Defects

One of the most worrying of all side effects of CellCept and MyFortic is the risk of birth defects. Evidence from adverse event reports collected by the FDA show that women taking one of these during the first trimester of pregnancy have an increased risk of giving birth to a child with one or more birth defects. They are also at a greater risk of having a miscarriage.

The risk is considered to be strong enough that the FDA requires both medications carry a black box warning on their information labels. The agency also requires that women taking either medication register on a special website to that information about pregnancy, miscarriages, and birth defects can be collected.

The current recommendation for women using CellCept or MyFortic is to take two pregnancy tests before starting them, and to use at least two forms of birth control during use and for at least six weeks after using them. These drugs have the potential to lower the effectiveness of hormone-based contraceptives, which is why two forms of birth control are strongly recommended.

Infections

Because mycophenolic acid suppresses the immune system, infections are a serious potential side effect. While taking the medication, the immune system is purposely weakened and is not able to fully fight off all pathogens. People on this drug are susceptible to any infection to which they are exposed from yeast and fungal infections to viral infections like shingles, herpes, or the flu, and bacterial infections.

Some of the more serious infections that patients on mycophenolic acid may experience as a side effect include hepatitis B and C. They may also be at risk for cytomegalovirus, which can lead to life-threatening blood infections, and BK virus which can cause kidney damage and failure.

Pure Red Cell Aplasia

A less common, but serious potential side effect of both CellCept and MyFortic is a condition called pure red cell aplasia. This possible side effect was not reported to the FDA until 2009. It is a type of anemia in which too few blood cells are present in the body, and is related to the suppression of the immune system. Pure red cell aplasia can cause severe lethargy and fatigue and is characterized by pale skin. Patients can recover from this by stopping use of mycophenolic acid, but this is not always an option.

Progressive Multifocal Leukoencephalopathy

In 2008 the FDA issued a safety statement about the possibility that mycophenolic acid could trigger progressive multifocal leukoencephalopathy, or PML. This is a rare, but serious infection caused by the JC virus. Many people are exposed to the virus, but typically only people with compromised immune systems are vulnerable to an infection, including those taking a drug like mycophenolic acid.

The infection attacks the central nervous system, specifically the insulating material that surrounds nerve cells. Symptoms of an infection may include poor coordination and clumsiness, loss of memory, trouble speaking, changes in vision, and leg weakness. The infection can be fatal. Cases of PML in patients using mycophenolic acid are most common when the drug is used for off-label treatments, such as for lupus or other autoimmune disorders. There is no fool-proof treatment for PML. The most common strategy is to restore the function of the immune system, but for patients with an organ transplant, that can be problematic.

Other CellCept Side Effects

These very serious side effects of CellCept and MyFortic are not the only potential downsides to using the medications. There are also more common side effects, which are not as severe or life-threatening. These include edema, or swelling, which most often occurs in the feet, ankles, and lower legs, stomach pain, vomiting, diarrhea, and high blood pressure. Children may also experience fevers, sore throats and low white and red blood cell counts.

An allergic reaction is also a potential side effect and for some people this reaction may become severe or life-threatening. Signs of an allergic reaction include a rash or itchiness, swelling in the face, mouth, and throat, difficulty swallowing, and difficulty breathing. This has to be treated as an emergency or it will be fatal. Allergic reactions may be less severe, but it is always something to be taken seriously as it can worsen and become life-threatening with little warning.

Of all the potential side effects of CellCept and MyFortic, birth defects are most likely to lead to lawsuits in the future. The risk was not made public until years after the drugs were on the market, and some women believe Roche and Novartis were negligent in not warning them earlier of the risks. The companies have already been found to be guilty of providing kickbacks to sell more of their products and have paid billions of dollars in fines. Lawsuits and settlement fees may be added to this in the future.