Sarep­ta's Ex­ondys 51 is not cost-ef­fec­tive, nor par­tic­u­lar­ly ben­e­fi­cial for DMD pa­tients — ICER

Par­ents with chil­dren suf­fer­ing from Duchenne mus­cu­lar dy­s­tro­phy (DMD) — a rare, pro­gres­sive mus­cle wast­ing dis­ease that dis­pro­por­tion­ate­ly af­fects young boys — cheered in 2016 when Sarep­ta Ther­a­peu­tics’ Ex­ondys 51 (eteplirsen) was con­tro­ver­sial­ly ap­proved by the FDA, un­der pres­sure from pa­tient ad­vo­cates and de­spite stiff op­po­si­tion from with­in and out­side the agency. Crit­ics lam­bast­ed the agency’s de­ci­sion, cit­ing in­suf­fi­cient da­ta — and now, an in­creas­ing­ly in­flu­en­tial ICER has con­curred: The un­der­ly­ing ev­i­dence sup­port­ing the drug is sparse, and giv­en its cur­rent pric­ing, it is cer­tain­ly not cost-ef­fec­tive.

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Venture & Research Associate

Alexandria Real Estate Equities

San Francisco, CA, USA