Posted from: Tuesday, December 11, 2018 - 05:14 PM - Present

Gamifant Approved to Treat Rare Immune Disease

November 20, 2018 – The U.S. FDA has approved Gamifant® (emapalumab-lzsg), manufactured by Sobi, to treat adult and pediatric (newborn and older) patients who have primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance to conventional HLH therapy. This marks the first time the FDA has approved a drug specifically for the treatment of HLH.

Primary HLH is a rare, inherited immune condition with a high rate of mortality. A genetic mutation causes the body to produce excess interferon gamma, which activates an overabundance of immune cells. The result is chronic inflammation that leads to eventual organ damage. The disease typically manifests during the first year of life, and can be fatal within a few months if left untreated. Treatments often aim to stabilize patients and halt the inflammatory process, after which a bone marrow transplant may be performed to help reprogram the immune system.

The recommended starting dose with Gamifant is 1mg/kg administered as an intravenous infusion over the course of one hour, twice per week. If needed, the dosage can be increased to a maximum of 10mg/kg. Patients should receive dexamethasone concomitantly, and continue on Gamifant until they receive a bone marrow transplant or until treatment becomes intolerable. In a clinical study, 63% of patients who received Gamifant responded to treatment. Of this group, 70% stabilized sufficiently to undergo a bone marrow transplant.

Sobi plans to launch the product during the first quarter of 2019. Pricing plans have not been announced.

Last Updated Monday, January 21, 2019 - 08:14 AM.