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Sobi’s $50M bet on a rare disease drug pays off with a quick FDA nod

Just 4 months after Sobi paid Novimmune $50 million in cash — and put $400 million up in milestones — for a rare disease drug called emapalumab, the FDA has followed up with a quick OK on marketing.

The drug is used to treat hemophagocytic lymphohistiocytosis, or HLH, which is an autoimmune condition in which cells become overactive, releasing molecules that spur inflammation. The FDA notes that symptoms include “fever, enlarged liver or spleen and decreased number of blood cells.”

The drug will now be marketed as Gamifant.

This is a disease that primarily afflicts children, warranting a priority review at the agency as the first treatment that is specifically designed for this condition. In a small study 63% of patients responded to the drug in some fashion, with 70% going on to a needed stem cell transplant.

The drug was also granted the FDA’s breakthrough drug designation, giving it a quick ride through the regulatory process. And the EMA has been equally obliging, offering its own assurances of a swift review and decision in 2019.

Not surprisingly, Sobi execs have done their math on peak sales potential. Surprisingly, they told everybody: A bit more than $300 million a year. And there’s a possibility they can expand the deal to include new assets around this drug.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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