Bio­Marin gets its dwarfism drug in the FDA's door — but can they change reg­u­la­tors’ minds about a longer fol­low up?

Bio­Marin’s dwarfism drug has se­cured a place in the FDA’s re­view line, but dis­agree­ments with reg­u­la­tors on just how long the fol­low-up pe­ri­od should be are threat­en­ing to put the biotech in an un­easy po­si­tion.

We’ll find out what the agency thinks about vosori­tide by Au­gust 20, 2021. But up­on ac­cept­ing the fil­ing the FDA made it abun­dant­ly clear that a pan­el of out­side ex­perts had rec­om­mend­ed two-year con­trolled tri­als across dif­fer­ent age groups. Bio­Marin’s da­ta pack­age, mean­while, fea­tures chil­dren aged 5 to 18. And in­ves­ti­ga­tors on­ly fol­lowed them for a year.

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