The negative opinion was given after the re-examination requested by Sobi after the initial opinon in
'This recommendation by the CHMP is disappointing given the significant unmet medical need which exists for patients with pHLH who have no approved therapies in
About primary HLH
Primary HLH is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. In spite of some treatment advances, there continues to be a very high unmet medical need in particular in patients that have failed conventional therapy as there are no approved treatment options outside the US. In the US, emapalumab is the first therapy approved by the
About emapalumab
Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFN). In the US, emapalumab is indicated for the treatment of adult and paediatric (new-born 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). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT).
About SobiTM
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across
This information is information that
Contact:
Paula Treutiger
Tel: + 46 733 666 599
Email: paula.treutiger@sobi.com
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