ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarep­ta and the FDA as the US drug price watch­dog con­cludes that as cur­rent­ly priced, their re­spec­tive new treat­ments for Duchenne mus­cu­lar dy­s­tro­phy are de­cid­ed­ly not cost-ef­fec­tive.

The fi­nal re­port — which ce­ments the con­clu­sions of a draft is­sued in May — in­cor­po­rates the opin­ion of a pan­el of 17 ex­perts ICER con­vened in a pub­lic meet­ing last month. It al­so based its analy­sis of Em­flaza (de­flaza­cort) and Ex­ondys 51 (eteplirsen) on up­dat­ed an­nu­al costs of $81,400 and over $1 mil­lion, re­spec­tive­ly, af­ter cit­ing “in­cor­rect” low­er num­bers in the ini­tial cal­cu­la­tions.

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