Swedish Orphan Biovitrum AB (publ) (Sobi™) announced that the Committee for Medicinal Products for Human use (CHMP) has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis (HLH) in children under 18 years of age in Europe.
Given the significant unmet medical need that emapalumab addresses in patients with primary HLH with no approved treatments in Europe, Sobi will be requesting a re-examination by the CHMP with an expected opinion by end of year 2020.
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 FDA for primary HLH. Over 100 patients have been treated in the US and the benefit/risk profile continues to be favourable.