FDA Okays First Treatment for Rare Inflammation Disorder

The FDA approved Gamifant (emapalumab-lzsg) as the first-of-its kind treatment for a rare and often fatal disorder.

Primary haemophagocytic lympohistiocytosis occurs when the body makes too many activated immune cells, which often attack the liver, spleen or blood cells.

Orphan Biovitrum and Novimmune reported the drug was effective at treating refractory, recurrent or progressive versions of the disease in a phase II/III trial. At least 20 percent of the patients in the trial were made healthy enough to receive a stem cell transplant.