Fibrous Dysplasia of Bone

Sobi’s Gamifant was previously approved by the US regulator in 2018. © Swedish Orphan Biovitrum AB. The US Food and Drug Administration has accepted Sobi’s supplemental biologics licence application for Gamifant (emapalumab-Izsg) for haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with Still’s disease. The application, which seeks approval for use in both adult and paediatric individuals who show an insufficient response or are not tolerant to glucocorticoids, or with recurrent MAS, has received priority review status. A Prescription Drug User Fee Act date was set by the agency for 27 June 2025. The sBLA is supported by pooled data outcomes from the EMERALD and NI-0501-06 studies, which enrolled a total of 39 subjects. 53% of subjects achieved a complete response by week 8 and 85% at any time during the studies. Sobi chief medical officer and research and development head Lydia Abad-Franch stated: “HLH/MAS in Still’s disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure. “Gamifant (emapalumab-Izsg) selectively neutralises interferon-gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients.” The therapy was previously approved by the US regulator in 2018 to treat primary HLH in adult and paediatric patients, including newborns, with refractory, recurrent or progressive conditions, or those who are not tolerant to conventional HLH therapies. A critical complication of rheumatic conditions, HLH/MAS is most commonly observed in Still’s disease, including systemic juvenile idiopathic arthritis and adult-onset Still’s disease. It is marked by severe inflammation, presenting with symptoms such as persistent high fever, elevated levels of ferritin, cytopenias, hepatosplenomegaly and coagulopathies. The company received marketing authorisation from the European Commission in June 2024 for Altuvoct (efanesoctocog alfa), a treatment designed for haemophilia A patients to manage bleeds and provide perioperative prophylaxis.

STOCKHOLM, Feb. 27, 2025 /PRNewswire/ -- Sobi® (STO: SOBI), today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-Izsg) for use in adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. The application was granted Priority Review with a PDUFA date of June 27, 2025. HLH/MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still's disease including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). HLH/MAS is a rare systemic disorder of hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. "HLH/MAS in Still's disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure," said Lydia Abad-Franch, MD, MBA, Head of R&D and Chief Medical Officer at Sobi. "There is no approved therapy for HLH/MAS today. Gamifant® (emapalumab-Izsg) selectively neutralizes interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients." The application is based on results from pooled data from two studies which enrolled a total of 39 patients, the EMERALD (NCT05001737) and the NI-0501-06 (NCT03311854) studies. Fifty-three percent of patients had a complete response at Week 8 and 85% had a complete response at any time during the studies. Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. Gamifant® (emapalumab-Izsg) a monoclonal antibody that binds and neutralises interferon gamma (IFN-y), was approved by the FDA in 2018 for the treatment of adult and paediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinemia, possibly progressing to multiple organ failure and death. MAS is classified as a form of hemophagocytic lymphohistiocytosis (HLH). About Gamifant® (emapalumab-lzsg) Gamifant® (emapalumab-lzsg) is an anti-interferon gamma (IFN-γ) monoclonal antibody that binds to and neutralises IFN-γ. In the USA, Gamifant® (emapalumab-lzsg) is the first and only treatment approved by the FDA for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is 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 is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant® (emapalumab-lzsg) is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Sobi® (STO: SOBI), today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-Izsg) for use in adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still’s disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.The application was granted Priority Review with a PDUFA date of June 27, 2025. HLH/MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease (AOSD). HLH/MAS is a rare systemic disorder of hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. “HLH/MAS in Still’s disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure,” said Lydia Abad-Franch, MD, MBA, Head of R&D and Chief Medical Officer at Sobi. “There is no approved therapy for HLH/MAS today. Gamifant® (emapalumab-Izsg) selectively neutralizes interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients.” The application is based on results from pooled data from two studies which enrolled a total of 39 patients, the EMERALD (NCT05001737) and the NI-0501-06 (NCT03311854) studies. Fifty-three percent of patients had a complete response at Week 8 and 85% had a complete response at any time during the studies. Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. Gamifant® (emapalumab-Izsg) a monoclonal antibody that binds and neutralises interferon gamma (IFN-y), was approved by the FDA in 2018 for the treatment of adult and paediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. About macrophage activation syndrome (MAS)Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease (AOSD). MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinemia, possibly progressing to multiple organ failure and death. MAS is classified as a form of hemophagocytic lymphohistiocytosis (HLH). About Gamifant® (emapalumab-lzsg)Gamifant® (emapalumab-lzsg) is an anti-interferon gamma (IFN-γ) monoclonal antibody that binds to and neutralises IFN-γ. In the USA, Gamifant® (emapalumab-lzsg) is the first and only treatment approved by the FDA for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is 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 is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant® (emapalumab-lzsg) is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. We provide access to innovative treatments that transform life for people. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.