US FDA Fast Tracks Sobi's Emapalumab-lzsg for Macrophage Activation Syndrome

On May 24, 2024, Sobi North America, the North American branch of Swedish Orphan Biovitrum AB, announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg. This designation aims to accelerate the development and review of medications designed to treat serious conditions and address unmet medical needs. Emapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody currently being investigated as a potential treatment for Macrophage Activation Syndrome (MAS). This antibody works by binding to both free and receptor-bound Interferon gamma (INFγ), thereby neutralizing its biological activity.

MAS is a severe and potentially life-threatening complication often associated with rheumatic diseases, predominantly found in Still’s disease, including systemic juvenile idiopathic arthritis (sJIA). Interferon gamma plays a crucial role in MAS development. MAS is considered a form of hemophagocytic lymphohistiocytosis (HLH), characterized by symptoms such as fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities, and elevated ferritin levels. Without prompt diagnosis and treatment, MAS can rapidly lead to multiple organ failure and death.

Emapalumab-lzsg stands out as the only FDA-approved medication in the United States for treating primary HLH, a rare and often fatal hyperinflammatory syndrome if not diagnosed and managed swiftly. The approval, granted in 2018, was based on data from phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab-lzsg is indicated for both pediatric (newborn and older) and adult patients suffering from primary HLH who exhibit refractory, recurrent, or progressive disease or intolerance to standard HLH therapies. This medication is administered via intravenous infusion twice a week until hematopoietic stem cell transplantation (HSCT).

The efficacy and safety of emapalumab-lzsg are being further evaluated in a phase 3 study focusing on patients with MAS associated with Still’s disease or systemic lupus erythematosus (SLE). This study, known as EMERALD (NCT05001737), is crucial for further understanding the potential benefits and risks of the treatment in these specific conditions.

Sobi North America operates as the North American affiliate of the biopharmaceutical company Sobi, which is dedicated to transforming the lives of individuals with rare and debilitating diseases. Their product portfolio includes various approved treatments concentrating on immunology, hematology, and specialty care. The North American team, headquartered in the Boston area with Canadian headquarters in Toronto, consists of field sales, medical, and market access representatives throughout North America. The team has demonstrated a strong record of commercial success.

Sobi is an international biopharmaceutical company with a mission to provide innovative treatments for rare and severe diseases. The company employs around 1,800 people across multiple regions, including Europe, North America, the Middle East, Asia, and Australia. In 2023, Sobi reported revenue of SEK 22.1 billion, and its shares are listed on Nasdaq Stockholm under the ticker symbol STO:SOBI. 

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