First Treatment for Hemophagocytic Lymphohistiocytosis is Approved by the FDA

According to a story from BioSpace, the biopharmaceutical company Sobi and the biotechnology company NovImmune SA have recently announced that the US Food and Drug Administration (FDA) has approved Gamifant, making it the first officially approved therapy for primary hemophagocytic lymphohistiocytosis (HLH). The treatment is meant for patients with recurrent or refractory progressive disease or for patients who are intolerant to conventional therapeutic approaches.

About Hemophagocytic Lymphohistiocytosis (HLH)

Hemophagocytic lymphohistiocytosis (HLH) is a disorder of the blood which tends to occur in kids more frequently than adults. This life-threatening condition is characterized by severe hyperinflammation. This is caused by the abnormal proliferation of white blood cells which release a high concentration of cytokines, signaling an inflammatory reaction. The disease is caused by mutations in genes which play a role in the destruction of targeted cells by the immune system. HLH can also occur secondarily alongside a number of autoimmune diseases and blood cancers like leukemia. Symptoms include jaundice, rash, enlarged liver, spleen, and lymph nodes, and fever. Prompt treatment is often necessary and may include high doses of steroids, chemotherapy agents like etoposide, IVIg, and other drugs that suppress the immune system. HLH has a survival rate of only 50 percent. Secondary HLH can often worsen outcomes for patients dealing with other diseases. To learn more about HLH, click here.

About Gamifant

The approval of Gamifant for public use is based on results from a Phase II/III clinical trial which consisted for 34 patients with HLH. In this study, treatment with Gamifant alongside dexamethasone resulted in a 63 percent response rate. The approval of Gamifant is the first advancement in the treatment of HLH in almost 25 years. 70 percent of the patients in the study were able to stabilize enough to become eligible for stem cell transplant, which can also treat the disease.

Gamifant utilizes a unique mechanism of action as an antibody which inhibits the activity of interferon gamma, which plays a major role in the HLH disease mechanism. The drug is expected to become publicly available in early 2019. This new therapy has the potential to significantly improve outcomes for patients with HLH.


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