Updated: BioMarin’s hemophilia A gene therapy Roctavian wins FDA approval after delay and rejection, priced at $2.9M

The FDA ap­proved the first gene ther­a­py to treat the ge­net­ic bleed­ing dis­or­der he­mo­phil­ia A on Thurs­day, cap­ping a tur­bu­lent reg­u­la­to­ry saga for Bio­Marin, which will now turn to com­mer­cial­iz­ing the one-time treat­ment.

Se­cur­ing the ap­proval for Roc­ta­vian was no easy feat. There was a sur­prise re­jec­tion in 2020 and a re­view ex­ten­sion ear­li­er this year, and now Bio­Marin has to sell the ther­a­py, which it hopes can grow in­to a key block­buster drug that can help dou­ble com­pa­ny rev­enues to $4 bil­lion to $5 bil­lion by the mid­dle of the decade. The drug­mak­er record­ed rev­enues of about $2.1 bil­lion last year.

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