Groundbreaking Study Reveals New Hope for Patients with Secondary HLH/Macrophage Activation Syndrome: Study Results Published in Annals of Rheumatic Diseases

Sobi North America and Swedish Orphan Biovitrum AB (Sobi®) have jointly announced the publication of findings from a pivotal phase 2 study in the Annals of Rheumatic Diseases.

The study evaluated the efficacy and safety of emapalumab, an anti-interferon-gamma monoclonal antibody, in treating Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still’s Disease (AOSD) patients who developed secondary hemophagocytic lymphohistiocytosis (sHLH)/Macrophage Activation Syndrome (MAS) following an inadequate response to high-dose glucocorticosteroids. The results of this study may potentially provide a new therapeutic option for these patients.

Sobi’s EMERALD phase 3 study is now underway to further evaluate emapalumab in patients with sJIA or AOSD and sHLH/MAS, after the promising results of a recent published study. In the trial, 14 patients who were unresponsive to high-dose glucocorticosteroids received emapalumab, and experienced MAS remission within 25 days on average. All 14 patients completed the trial and have entered long-term follow-up, and are alive and well at this time.

Dr. Fabrizio De Benedetti, Head of the Division of Pediatric Rheumatology and Head of the Laboratory of ImmunoRheumatology at Bambino Gesù Children’s Hospital in Rome, Italy, is enthusiastic about the potential for emapalumab to become a targeted treatment for sJIA or AOSD with sHLH/MAS who have failed high-dose glucocorticoids. He believes that the results of this study represent a major breakthrough in treating this rare disease and its unmet medical need.

Systemic Hyperinflammation and Macrophage Activation Syndrome (sHLH/MAS) is a rare and life-threatening condition that can be triggered by underlying rheumatologic diseases such as sJIA. It is classified as a secondary form of Hemophagocytic Lymphohistiocytosis (HLH) and is characterized by an excessive activation and expansion of T cells and macrophages.

Recent research has identified Interferon gamma (IFNγ) as a key factor in driving the hyperinflammation that causes sHLH/MAS, highlighting the importance of understanding the mechanisms of this condition.

The results of a study recently published in the Annals of Rheumatic Diseases are promising for patients with sJIA or AOSD who have had an inadequate response to high dose glucocorticoid treatment, and have developed sHLH/MAS.

According to Dr. Alexei Grom of Cincinnati Children’s Hospital Medical Center, the ability of emapalumab to achieve MAS remission is an important breakthrough for these individuals, as there is a great need for better treatments.

Sobi is delighted to announce the publication of new clinical data revealing the potential of emapalumab to benefit patients with secondary HLH. We remain dedicated to assessing emapalumab as a potential treatment option for those affected by this severe condition. If the ongoing EMERALD phase 3 study in sHLH/MAS demonstrates a benefit, we plan to submit a supplemental Biologics License Application.

Gamifant®, the United States’ first treatment for primary hemophagocytic lymphohistiocytosis (HLH), is now available for adult and pediatric patients alike. Whether facing refractory, recurrent or progressive disease, or intolerance with conventional HLH therapy, Gamifant® offers a proven solution with proven results. It’s the answer for patients seeking relief from the symptoms of HLH.

Gamifant has yet to receive the stamp of approval from the U.S. Food and Drug Administration for its use in treating sHLH/MAS, but its potential to help patients suffering from this condition is undeniable. With the right support, this innovative technology could be a game-changer in the fight against sHLH/MAS.

About macrophage activation syndrome (MAS)

Macrophage activation syndrome (MAS) is a life-threatening complication of systemic juvenile idiopathic arthritis (sJIA), a rare and serious auto-inflammatory disorder. Symptoms include daily spiking fever, a transient rash, arthritis, and swelling of the liver, spleen, and other organs.

MAS is clinically characterized by fever, organ swelling, blood cell deficiencies, blood clotting problems, and high levels of iron in the blood, which can lead to multiple organ failure and death. It is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH).

About emapalumab-lzsg

Emapalumab-lzsg is a revolutionary breakthrough for those suffering from primary hemophagocytic lymphohistiocytosis (HLH)! Approved by the FDA in 2018, this fully human, anti-IFNγ monoclonal antibody is the first and only medicine to be approved in the US for this rare, potentially fatal syndrome of hyperinflammation.

It is administered through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT) and has been shown to be effective and safe in phase 2/3 studies.

What’s more, its efficacy and safety are currently being evaluated in a phase 3 study for other inflammatory conditions such as MAS in Still’s disease or systemic lupus erythematosus (SLE). Emapalumab-lzsg is a life-saving option for those with primary HLH and gives hope to those suffering from other inflammatory conditions.

U.S. Indication for Gamifant® (emapalumab-lzsg)

Gamifant® (emapalumab-lzsg) is an innovative treatment for adult and pediatric patients suffering from primary hemophagocytic lymphohistiocytosis (HLH). It has been proven to be effective in treating refractory, recurrent, or progressive cases of HLH, as well as cases where patients have shown intolerance to conventional HLH therapy. This unique solution works by blocking the interferon gamma (IFNγ) and providing relief for those suffering from this serious condition.

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