FDA approves Ipsen’s ultra-rare disease drug palovarotene

The FDA gave a nod of ap­proval to Ipsen’s treat­ment for the ul­tra-rare dis­ease fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va though reg­u­la­tors have ex­pressed con­cerns at least twice about the safe­ty and ef­fi­ca­cy of the drug.

“For the first time doc­tors have an ap­proved med­i­cine avail­able to them, shown to re­duce the for­ma­tion of new, ab­nor­mal bone growth, known as het­ero­topic os­si­fi­ca­tion (HO), which caus­es de­bil­i­tat­ing mo­bil­i­ty chal­lenges and has a dev­as­tat­ing im­pact on the lives of peo­ple with FOP,” Howard May­er, head of R&D at Ipsen, said in a state­ment.

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