Speedy re­view and no ad­comm for Bio­Mar­in's pi­o­neer­ing he­mo­phil­ia gene ther­a­py

Bio­Marin’s keen­ly an­tic­i­pat­ed he­mo­phil­ia A gene ther­a­py — which CEO JJ Bi­en­aimé en­vi­sions pric­ing at an eye-wa­ter­ing $2 mil­lion to $3 mil­lion a shot — has se­cured red car­pet treat­ment at the FDA.

The ther­a­py, fond­ly called val­rox, has won pri­or­i­ty re­view — and there the agency has in­di­cat­ed there are no plans for an ad­vi­so­ry com­mit­tee meet­ing. The US reg­u­la­tor’s fi­nal de­ci­sion is ex­pect­ed by Au­gust 21. If ap­proved, it will be the pi­o­neer­ing gene ther­a­py for he­mo­phil­ia.

“While the com­pa­ny…in­di­cat­ed that FDA does not cur­rent­ly plan on con­ven­ing an ad­vi­so­ry com­mit­tee meet­ing to dis­cuss the mer­its of the ap­pli­ca­tion, we cau­tion that the pos­si­bil­i­ty of such still re­mains as the agency digs in­to its re­view, which could be in­ter­pret­ed neg­a­tive­ly by the Street,” BMO Cap­i­tal Mar­ket’s George Farmer wrote.

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