What are Off-Label Drugs for Thyroid Eye Disease?

Written by: Katie Murphy | Last reviewed: June 2022 | Last updated: July 2022

Unfortunately, rare diseases like thyroid eye disease (TED) may have fewer approved treatment options than more common diseases. Your TED specialist may discuss treatment options with you that include off-label drugs.

An off-label drug is one that is used to treat a different condition than the one it was approved by the U.S. Food and Drug Administration (FDA). This also means the FDA has not yet determined the drug is safe and effective when used the way you are taking it.1

Using drugs off-label is common in rare diseases. This is because there often are not many drugs available to treat these conditions. If you and your doctor decide to use an off-label drug, it is important to understand what off-label means.1

Off-label does not mean the drug is unsafe to take. Often, it means there has not been an official clinical trial to test the drug for this specific rare disease. You should always talk to your doctor about any questions you have about your treatments.1

There are several off-label drugs used for TED.

Cosentyx® (secukinumab)

Cosentyx® (secukinumab) is a biologic drug that has been used for other autoimmune conditions. Biologics are drugs made in a lab from living cells. These cells can come from blood, proteins, viruses, or tissue. This process turns the cells into drugs that can treat TED.2

As of 2021, Cosentyx is not an approved treatment for TED. Clinical trials are still underway to prove its efficacy and safety for treating the disease.2

CellCept® (mycophenolate mofetil, or MMF)

Mycophenolate mofetil, also called CellCept® or MMF, is a drug sometimes used to treat TED. Limited studies have shown effectiveness and safety for use in TED. A 2021 study showed that MMF may be an effective second-line treatment for moderate to severe cases of TED, as well as sight-threatening cases. However, more studies involving larger groups of people over a longer period of time are needed to prove that MMF is a good treatment option in TED.3,4

Rituxan (rituximab)

Rituxan (rituximab) is a biologic drug and monoclonal antibody. This is a lab-made antibody that targets a certain antigen. An antigen is a molecule that triggers an immune response in the body. Rituxan is an antibody that attaches to B cells, a type of white blood cell in your body that can trigger inflammation.5

Though doctors do not know exactly how Rituxan works in TED, it is thought to help prevent attacks by blocking B cells and decreasing inflammation.5

Actemra® (tocilizumab)

Actemra® (tocilizumab) is a laboratory-made antibody to the IL-6 receptor, which has been shown to play a role in the inflammation TED can cause. Several studies have shown that Actemra may improve the symptoms of TED, especially for those who have not had success with steroid drugs. However, more studies are needed to prove its safety and efficacy in TED.6,7

As of 2021, some drugs that were once thought to benefit TED are no longer conducting clinical trials due to the unwanted side effects these drugs may cause.

Talk to your TED specialist about the treatment options that are best for you. Approved and off-label drugs have risks and benefits. Having a conversation with your doctor can help you decide which treatment path you take.

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