311 Medicines in the Pipeline for Autoimmune Diseases: Raymond Rupert MD. MBA.

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The 311 medicines in development for patients with autoimmune diseases use exciting new approaches aided by the growing scientific understanding of many of these diseases. In addition, medicines already approved to treat one autoimmune disease are being studied for applicability in treating additional diseases.

Some of the medicines in the pipeline for autoimmune diseases include: LUPUS (SYSTEMIC LUPUS ERYTHEMATOSUS) A monoclonal antibody medicine in development for the treatment of lupus acts against a protein in the body that is thought to play a key role in the development of the disease. The protein helps regulate the activity of the immune system. By inhibiting the activity of the protein, the medicine is expected to halt the development of lupus.

PSORIASIS Several monoclonal antibody medicines are in development for psoriasis that target a specific subunit of a receptor gene. The monoclonal antibodies bind to and neutralize the gene, which is thought to play a key role in autoimmune inflammatory processes that have been linked to many chronic autoimmune diseases, including psoriasis.

SJÖGREN’S SYNDROME A monoclonal antibody medicine in development for the treatment of Sjögren’s syndrome binds to a protein in the body and prevents its interaction with stimulators on immune cells called activated T-cells. Preventing the interaction between the protein and the stimulator is thought to prevent increased immune cell responses associated with autoimmune diseases, such as Sjögren’s.


The diagnosis of autoimmune diseases is challenging for several reasons. A lack of basic knowledge about each disorder and how they are different from each other make it difficult to diagnose patterns, identify treatments and design clinical trials. Researchers are working to gain a better understanding of the causes, underlying biology and factors affecting disease progression of these disorders. Autoimmune diseases are complex and can share similar symptoms or affect similar parts of the body, making these conditions difficult to diagnose.

For example, early symptoms of several autoimmune diseases can include inflammation, achy muscles, fatigue and fever. And, if the skin is affected, it could be one of many autoimmune diseases, such as scleroderma and vitiligo. Additionally, many autoimmune diseases are characterized by symptoms that may occur in acute episodes; these episodes may ebb and change over time, making it difficult to determine the severity of the disease.


The lack of knowledge regarding the biology of autoimmune diseases makes it difficult to identify targets for clinical research. Before a new medicine can be approved by the FDA, researchers must demonstrate that the medicine is safe and effective. Evaluating the effects of new medicines in patients is done through clinical trials. Specific measures, called endpoints, are used to evaluate safety and efficacy; however, it is challenging to define viable measures and endpoints to determine how the disease is progressing and whether a medicine is effective.

Clinical trials for autoimmune diseases may also be delayed, as it is challenging to enroll patients with uncertain diagnoses or co-occurring diseases. In order to accelerate the development of new treatment options, biopharmaceutical researchers work closely with patients and members of the autoimmune disease community to develop innovative ways to assess disease activity.

For example, scoring systems, such as the systemic lupus erythematosus disease activity index (SLEDAI) were developed in order to find a way to quantify lupus disease activity so that researchers could measure the impact of new medicines in development. This innovative disease scale encompasses measures that are important for patients, including seizures, psychosis, hair loss and muscle weakness, as well as laboratory testing results, including urinalysis and blood cell levels, that can be disease indicators. Biological markers, or biomarkers, are also emerging as important new ways of detecting disease and monitoring disease progression.

Biomarkers are a measure or physical sign that can also be used to assess how the body is functioning. In patients with autoimmune diseases, these measures can indicate when the immune system is malfunctioning. Biomarkers may be used to diagnose a disease earlier, before irreversible damage has been done, and help determine if a medicine being evaluated in clinical trials is effective.

PHRMA Research Report  Sept 2016.