Swiss biopharmaceutical company Novimmune has received rare paediatric disease designation for its lead drug emapalumab from the US Food and Drug Administration (FDA) to treat patients with primary haemophagocytic lymphohistiocytosis (HLH).

HLH is a hyperinflammatory syndrome that involves uncontrolled and aberrant activation of the immune system and a life-threatening cytokine storm.

The disease results in non-remitting fever, reduction in the number of blood cells and impaired coagulation that potentially leads to death.

Discovered and developed by Novimmune, emapalumab is a fully human, anti-interferon-gamma (IFNγ) monoclonal antibody.

The biological activity of IFNγ is considered to have a major pathogenic role in HLH and is neutralised by emapalumab, the first targeted therapy for the rare paediatric disease.

Once the eligibility of emapalumab for the treatment of primary HLH is confirmed at the time of the FDA approval, the company will qualify for a rare paediatric disease priority review voucher.

"We are looking forward to working with the regulatory authorities to make this medicine available to patients with this devastating disease as soon as possible."

Novimmune chairman and chief executive Eduard Holdener said: “We are delighted to have been granted this special designation for our emapalumab programme.

“We are looking forward to working with the regulatory authorities to make this medicine available to patients with this devastating disease as soon as possible.

“This priority review voucher is also precious as it can be used to secure the priority review of another programme with the FDA and may be transferred between companies.”

HLH can occur in two forms, of which the primary HLH is genetically determined and is particularly diagnosed in infants and young children, and the secondary HLH is found on a background of a rheumatic / inflammatory disease, a malignancy or an infection.


Image: Very high magnification micrograph of haemophagocytic syndrome. Photo: courtesy of Nephron.