Sobi Receives US FDA Fast Track Designation for Emapalumab-lzsg being investigated in Macrophage Activation Syndrome
Drug ApprovalFast TrackClinical Result
May 24, 2024 - Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg being investigated as a potential therapeutic option in patients with Macrophage Activation Syndrome (MAS). Emapalumab is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. Emapalumab is currently in Phase lll development for MAS.
Fast track designation is designed to facilitate the development and expedite the review of medicines to treat serious conditions that may fill an unmet medical need.
MAS is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA). Interferon gamma (INFy) is a key cytokine in the development of MAS. MAS is classified as a form of haemophagocytic lymphohistiocytosis (HLH) and characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly rapidly progressing to multiple organ failure and death.
Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that can rapidly become fatal unless diagnosed and treated.
About emapalumab-lzsg
Emapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737).
U.S. Indication for Gamifant® (emapalumab-lzsg)
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.
About Sobi North America
As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa or at www.sobi.com.
About Sobi®
Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.
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