Near­ly two years af­ter their nar­row PhI­II fail­ure, Or­p­hazyme gets speedy look at the FDA

Or­p­hazyme’s bet that, de­spite miss­ing both pri­ma­ry end­points on its piv­otal study, they would find wel­com­ing ears at the FDA has paid off, for now.

The Dan­ish biotech an­nounced to­day that the FDA has ac­cept­ed the ap­pli­ca­tion for their ex­per­i­men­tal drug ari­mo­clo­mol in the rare neu­rode­gen­er­a­tive con­di­tion Nie­mann-Pick dis­ease Type C and giv­en it pri­or­i­ty re­view. Al­though drugs that fail piv­otal stud­ies rarely re­ceive a pri­or­i­ty look at the agency, the dis­tinc­tion should come with lit­tle sur­prise. Or­p­hazyme has sig­naled re­peat­ed­ly since the 2019 re­sults came out that the FDA viewed an ap­pli­ca­tion fa­vor­ably and, the FDA gave it break­through des­ig­na­tion that No­vem­ber.

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