Kept on hold at the FDA, Ipsen scraps a key tri­al and re­vamps R&D path for its trou­bled $1B-plus rare dis­ease drug

Af­ter get­ting slammed by a par­tial hold on safe­ty fears and flunk­ing a fu­til­i­ty test, Ipsen says it has de­cid­ed to re­vamp its R&D plans for their trou­bled de­vel­op­ment pro­gram for palo­varotene, scrap­ping a side­lined study while re­vis­ing its tri­al analy­sis in hopes of cap­tur­ing pos­i­tive da­ta.

The French biotech, which ac­quired the drug in their $1 bil­lion-plus Clemen­tia buy­out, paused a Phase III and Phase II tri­al fol­low­ing the fu­til­i­ty analy­sis. Ear­li­er, the FDA had im­posed a hold on dos­ing pa­tients un­der the age of 14 af­ter see­ing ev­i­dence that the drug de­signed to pre­vent over­growth of bone for cas­es of fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va might ac­tu­al­ly stunt chil­dren’s growth.

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