Swiss biotech Novimmune has submitted a marketing application in Europe seeking permission to market its lead compound emapalumab for the treatment of patients with primary Hemophagocytic Lymphohistiocytosis (HLH).
The condition, classed an orphan disease for which there is currently no treatment, is a clinical syndrome of hyperinflammation, driven by high interferon gamma production and characterised by severe hyperferritinemia, fever, severe cytopenia, coagulation defects and organomegaly.
HLH occurs as a familial autosomal recessive disorder or as an acquired, reactive condition. The former typically presents in paediatric patients, is lethal if untreated, and has a 40 percent mortality rate with current best available care.
The secondary form of the disease typically arises later in life, and is also linked with significant mortality.
Emapalumab is a fully human, anti-interferon-gamma monoclonal antibody discovered and developed by Novimmune, which neutralises the biological activity of IFNγ, and thus offers the first targeted therapy for this disease.
Novimmune chairman and chief executive Eduard Holdener said filing the drug’s marketing application “is a very important event for children suffering from HLH as it represents a big step towards emapalumab becoming available in the future in Europe.”
The firm is currently in the process of selling global commercialisation and development rights to emapalumab to Swedish Orphan Biovitrum (Sobi), with the transaction expected to close in the third quarter.