Sobi® today announced that the
Primary HLH is an ultra-rare, life-threatening, hyperinflammatory disorder, characterised by uncontrolled activation of the immune system. Morbidity and mortality are high due to lack of detection and non-specific symptoms, but if diagnosed early and appropriately treated, the inflammation and damage can be reduced. Gamifant represents a new treatment option for patients with progressive disease through a targeted mode of action1.
"We are very pleased to announce the approval by the NMPA of this important treatment for patients with primary HLH in
About primary haemophagocytic lymphohistiocytosis
Primary haemophagocytic lymphohistiocytosis (HLH) is an ultra-rare, rapidly progressive, often-fatal syndrome of hyperinflammation in which hyperproduction of interferon gamma (IFNy) is thought to drive immune system hyperactivation, ultimately leading to organ failures. Diagnosis is challenging due to the variability in signs and symptoms, which may include fevers, swelling of the liver and spleen, severe low red and white blood cell counts, bleeding disorders, infections, neurological symptoms, organ dysfunction and organ failure. Primary HLH can rapidly become fatal if left untreated, with median survival of less than two months. The immediate goal of treatment is to quickly control the hyperinflammation and to prepare for haematopoietic stem-cell transplant. The current conventional treatment prior to transplant includes steroids and chemotherapy and are not specifically approved to treat primary HLH.
About Gamifant®
Gamifant (emapalumab) is a monoclonal antibody that binds to and neutralises interferon gamma. Gamifant is the first and only medicine approved in the US and
References
- Merli P, Algeri M, Gaspari S, Locatelli F. Novel Therapeutic Approaches to Familial HLH (Emapalumab in FHL). Front Immunol. 2020
Dec 2 ;11:608492. doi: 10.3389/fimmu.2020.608492. PMID: 33424859; PMCID: PMC7793976.
Sobi®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.
Postal address SE-112 76
Phone: 46 8 697 20 00 www.sobi.com
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