Positive Data Available from Phase 4 Trial for Primary Immunodeficiencies


In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin subcutaneous human–klhw) for individuals living with primary humoral immunodeficiency (PIDD), which may also be called primary immunodeficiency (PI). There are multiple subtypes of PI, including common variable immunodeficiency disease (CVID), chronic granulomatous disease (CGD), and severe combined immunodeficiency disease (SCID).

XEMBFIY, administered subcutaneously, is a 20% solution of purified human immunoglobulin. It is designed to treat primary immunodeficiencies in people aged 2+. XEMBIFY should not be used in people who have previously had severe, systemic, or anaphylactic reactions to human immune globulin products. Learn more about XEMBIFY here.

Altogether, 27 participants enrolled in this study. The study aimed to identify the efficacy or potential of biweekly XEMBIFY dosing. Researchers found that the trial met its primary endpoint. People with PI who were treated with XEMBIFY every two weeks achieved non-inferiority in immunoglobulin levels when compared to people treated weekly with XEMBIFY. As a result, Grifols now suggests that patients could have more flexible treatment options. This biweekly dosing method has not yet been approved by the FDA, however, though it has been approved in Europe.

While XEMBIFY was found to be relatively safe and well-tolerated, there were some side effects associated with treatment. These included injection-site reactions (such as bruising, pain, swelling, scabbing, or itchiness), coughing, or diarrhea. If you are on Xembify and experiencing negative side effects, you can report those to the FDA via either www.fda.gov/medwatch or call 1-800-FDA-1088.

What is a Primary Immunodeficiency (PI)?

Primary immunodeficiency diseases are rare, inherited immune system disorders in which the immune system does not function properly, preventing the body from adequately fighting infections. These fall under the larger category of inborn errors of immunity. There are over 450 forms of PI and these cause increased susceptibility to infection. A primary immunodeficiency results from mutated genes.

Outside of severe and persistent infections, people living with a PI may experience symptoms such as inflammatory bowel disease, blood vessel inflammation, and a swollen spleen, liver, or lymph nodes. Since symptoms vary depending on the specific PI subtype, it can be difficult to diagnose primary immunodeficiencies.

Treatment often varies and can focus on fighting infections, identifying and treating the underlying cause, or making the immune system stronger. Though treatments are often effective in managing symptoms, there are still no cures for primary immunodeficiencies.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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