Off-Label Use of Biologics for Axial Spondyloarthritis

Some biologics are approved by the U.S. Food and Drug Administration (FDA) to treat axial spondyloarthritis (AxSpA). Others are used off-label. Off-label drugs may be used when someone has not responded well to standard treatments. If you and your doctor decide to use an off-label drug, it is important to understand what “off-label” means.

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

This 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 condition.1

How do biologics work for AxSpA?

Biologics are a kind of disease-modifying antirheumatic drug (DMARD) made from living cells. Like standard DMARDs such as hydroxychloroquine and methotrexate, biologics work to change the course of diseases. For instance, they can keep diseases from getting worse.2,3

Biologics have more precise aims than DMARDs that work in general throughout the body. Biologics address certain aspects of immune system function. This helps lessen inflammation and its harmful effects. With AxSpA, biologics help reduce pain and swelling in the joints and connective tissues of the pelvis and spine.2,3

Biologic DMARDs help to calm down the immune system. Some biologics do this by blocking proteins known as tumor necrosis factor (TNF) and TNF-alpha. TNF and TNF-alpha are overproduced in people with inflammatory conditions like AxSpA. Other biologic DMARDs block the interleukin 17 (IL-17) protein involved in immune responses. Decreasing the amount of these proteins in the body reduces inflammation and damage.2,3

Which biologics are used off-label for AxSpA?

Some biologics are used off-label to treat an initial type of AxSpA without evident bone damage. This form of the disease is known as non-radiographic AxSpA (nr-AxSpA). These TNF blockers are used off-label for nr-AxSpA:4-7

  • Adalimumab (Humira®)
  • Etanercept (Enbrel®)
  • Golimumab (Simponi Aria®)
  • Infliximab (Remicade®)

Research shows these biologics improve the symptoms of nr-AxSpA. One study found adalimumab reduced inflammation and improved life for people who did not respond to NSAIDs. Another study found people with nr-AxSpA got better with golimumab treatment. Etanercept also has been shown to help people with nr-AxSpA when NSAIDs did not work.8-10

Which biologics are approved to treat AxSpA?

A few biologics used off-label in the past are now approved to treat nr-AxSpA. Certolizumab pegol (Cimzia®), a TNF blocker, was the first to receive FDA approval in 2019. In 2020, the FDA also approved the IL-17 inhibitors secukinumab (Cosentyx®) and ixekizumab (Taltz®) to treat nr-AxSpA.10

These biologics are FDA-approved to treat another type of AxSpA called ankylosing spondylitis (AS):3-7,12-14

  • Adalimumab
  • Certolizumab pegol
  • Etanercept
  • Golimumab
  • Infliximab
  • Ixekizumab
  • Secukinumab

What are the possible side effects of off-biologics for AxSpa?

Side effects can vary depending on the biologic you are taking.

Biologic DMARDs have side effects. Since biologics suppress the immune system, severe infections can result. Taking biologics may lead to new infections or trigger old infections like tuberculosis or hepatitis. Biologic DMARDs may also increase the risk of cancer.2-7,12-14

Biologic DMARDs share many of the same possible side effects. The most common include:2-7,12-14

  • Upper respiratory infections
  • Allergies
  • Autoimmune responses
  • Problems with the blood, heart, liver, and nervous system

People who take biologics may also have reactions at the injection site. Itching, pain, redness, and swelling may occur. People who get infliximab infusions may also have blood pressure extremes, chest pain, chills, fever, headaches, and rash.2-7,12-14

These are not all the possible side effects of biologics. Talk to your doctor about what to expect or if you notice any changes that concern you during treatment.

How can I try off-label biologics?

If you would like to try using a biologic off-label, talk to your doctor. Tell them about all of your health history. Also talk about any medicine, supplements, and vitamins you take, including over-the-counter drugs. They can check for existing infections, pregnancy, and anything else that might stop you from taking a biologic.

Off-label drugs may not be approved or covered by your insurance, so your doctor will work with you to find the best and most affordable options for you.

After a review of your overall health, your doctor may suggest a biologic to suit you. They may even teach you how to inject it under the skin, if it is given this way. The 2 of you can work together to watch for side effects.

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Written by: Michelle Marie Hernandez | Last reviewed August 2021