Em­maus who? A biotech you nev­er heard of just won an his­toric drug OK for sick­le cell dis­ease

Af­ter pur­su­ing an R&D odyssey that stretched out more than 15 years, lit­tle Em­maus Life Sci­ences in Tor­rance, CA has just won an FDA OK for En­dari, the first new drug ap­proved for sick­le cell dis­ease in close to 20 years.

Op­er­at­ed by Yu­ta­ka Ni­ihara, Em­maus gained the back­ing of a large ma­jor­i­ty of out­side ex­perts brought in by the agency to re­view the drug. “Un­til now, on­ly one oth­er drug was ap­proved for pa­tients liv­ing with this se­ri­ous, de­bil­i­tat­ing con­di­tion,” not­ed the FDA’s Richard Paz­dur in mark­ing the OK.

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