First Drug for Rare Inflammation Syndrome Approved by FDA

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The FDA has approved the first drug for the treatment of a rare condition in which the body produces too many active immune cells, developed in a Swiss-Swedish partnership.

The antibody drug, emapalumab, is approved to treat primary hemophagocytic lymphohistiocytosis. This condition is a rare, often fatal syndrome in which the immune system produces too many activated immune cells, as well as inflammatory molecules such as interferon gamma, causing inflammation and organ damage.

Although bone marrow transplants can cure the condition, they can fail and often require pre-conditioning with chemotherapy, to which some patients do not respond.

Emapalumab, an antibody developed by Swedish biopharmaceutical Sobi and Swiss biotech company Novimmune, is the first FDA drug approved specifically for this rare condition. It reduces inflammation by binding to, and neutralizing, interferon gamma.

The FDA approved the drug based on favorable results from a Phase II/III trial of the drug in patients whose treatments had failed previously. In the results, 63% of the 34 enrolled patients responded to the drug, and 70% were able to receive bone marrow transplants.

The approval comes just four months after Sobi paid Novimmune €44M upfront for perpetual global rights to emapalumab, with another €353M due in additional payments over an eight-year period. For Sobi, this drug is likely worth the expense, with an estimated sales potential of over €242M annually.

As one of the best-funded biotechs in Europe, Novimmune is also collaborating with other big names. For example, it is running a Phase II trial of an antibody treatment for rheumatoid arthritis with Genentech and, with the UK-US company Tiziana Life Sciences, it is planning to enter its drug for Nonalcoholic Steatohepatitis into Phase II.

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