48. Gamifant

(Sobi)

Active ingredient: emapalumab
Disease: primary hemophagocytic lymphohistiocytosis
Peak sales estimate: $330 million
Approved: Nov. 20
Company: Sobi (licensed from Novimmune) 

The scoop: The FDA approval of Gamifant during Thanksgiving week was a nice, quick win for Sobi, which spent just shy of $500 million on the drug a few months prior from originator Novimmune. This was a $50 million upfront deal, with the rest backloaded in milestones. The drug, an interferon gamma blocking antibody, now has a U.S. license to treat certain patients with primary hemophagocytic lymphohistiocytosis (HLH). Sobi, the only company with this license, could see a relatively modest $330 million from the drug a year at peak. This ultra-rare condition is one in which the body’s immune cells do not work properly and can attack organs, including the liver, and it can be inherited, with symptoms typically arising within the first months or years of life. Given its rarity, the drug was studied in just one phase 2/3 trial of 27 very young patients (around 1 year old) with suspected or confirmed primary HLH, with data showing 63% of patients saw a response and 70% were able to proceed to stem cell transplant. — Ben Adams 

48. Gamifant

Suggested Articles

Yale scientists have combined the gene-editing system CRISPR with gene therapy to help the immune system find tough-to-spot tumor cells.

The planned deal hit a wall when a special committee set up by Eidos turned down BridgeBio’s third and final offer.

Shares in ReNeuron fell more than one-third after the release of top-line data at the start of the month but the biotech argued the data are positive.