FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last sum­mer, Sarep­ta CEO Doug In­gram told Duchenne MD fam­i­lies and in­vestors that the FDA’s shock re­jec­tion of their sec­ond Duchenne MD drug golodirsen was due to some con­cerns reg­u­la­tors raised about the risk of in­fec­tion and the pos­si­bil­i­ty of kid­ney tox­i­c­i­ty. But when pressed to re­lease the let­ter for all to see, he de­clined, ac­cord­ing to a re­port from Bio­Phar­maDive, say­ing that kind of move “might not look like we’re be­ing as re­spect­ful as we’d like to be.”

He went on to as­sure every­one that he hadn’t mis­rep­re­sent­ed the CRL.

But In­gram’s pub­lic re­marks didn’t in­clude every­thing in the let­ter, which — fol­low­ing the FDA’s sur­prise about-face and un­ex­plained ap­proval — has now been post­ed on the FDA’s web­site and broad­ly cir­cu­lat­ed on Twit­ter ear­ly Wednes­day.

The CRL rais­es plen­ty of fresh ques­tions about why the FDA abrupt­ly de­cid­ed to re­verse it­self and hand out an OK for a drug a se­nior reg­u­la­tor at the FDA be­lieved — 5 months ago, when he wrote the let­ter — is dan­ger­ous to pa­tients. It al­so puts the spot­light back on Sarep­ta $SRPT, which failed to launch a con­fir­ma­to­ry study of eteplirsen, which was on­ly ap­proved af­ter a heat­ed in­ter­nal con­tro­ver­sy at the FDA. El­lis Unger, di­rec­tor of CDER’s Of­fice of Drug Eval­u­a­tion I, notes that study could have clar­i­fied quite a lot about the ben­e­fit and risks as­so­ci­at­ed with their drugs — which can cost as much as a mil­lion dol­lars per pa­tient per year, de­pend­ing on weight.

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