For­mer CBER chief Pe­ter Marks says that a de­ci­sion to pause ship­ments of Sarep­ta’s gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy “makes sense at this time,” and that there could be broad­er is­sues with as­pects of the treat­ment.

Marks, who left the FDA ear­li­er this year, con­tro­ver­sial­ly helped push Sarep­ta’s ther­a­py to ap­proval in 2024, over­rul­ing two of his top deputies who felt the treat­ment should be re­ject­ed and that the com­pa­ny should run an ad­di­tion­al clin­i­cal tri­al.

“Giv­en the re­cent de­vel­op­ments, a pause to re­view the sit­u­a­tion with Sarep­ta specif­i­cal­ly and the safe­ty of sys­tem­at­ic high dose AAV in gen­er­al makes sense at this time,” Marks told End­points News. “It is pos­si­ble that the man­u­fac­tur­ing com­plex­i­ty and dos­ing is­sues are larg­er than just this one pro­gram.”

The FDA will ask Sarep­ta to stop ship­ping its Duchenne mus­cu­lar dy­s­tro­phy treat­ment Ele­v­idys, an HHS of­fi­cial said, in what could be a fi­nal blow to the com­pa­ny's hopes for the prod­uct fol­low­ing two pa­tients' deaths.

The HHS of­fi­cial spoke on con­di­tion of anonymi­ty. News of the re­quest was re­port­ed ear­li­er by Reuters. Sarep­ta did­n't re­spond to a re­quest for com­ment.

The stock SRPT was down 38% on Fri­day af­ter­noon, and has now lost more than 90% of its val­ue in the last 12 months.

Since last month, the FDA has been in­ves­ti­gat­ing the deaths of two pa­tients who had re­ceived the ther­a­py and then gone in­to liv­er fail­ure. The com­pa­ny had al­ready stopped dis­trib­ut­ing the gene ther­a­py for use in pa­tients who are non-walk­ing, and are typ­i­cal­ly old­er and in a more ad­vanced phase of the dis­ease.

The FDA is­sued a com­plete re­sponse let­ter to Roche’s Columvi, say­ing the da­ta that the com­pa­ny pro­vid­ed for the la­bel ex­pan­sion aren't suf­fi­cient.

Roche’s Genen­tech had been seek­ing ap­proval for Columvi in com­bi­na­tion with gem­c­itabine and ox­ali­platin for pa­tients with re­lapsed/re­frac­to­ry dif­fuse large B-cell lym­phoma who are not el­i­gi­ble for au­tol­o­gous stem cell trans­plant. It an­nounced the re­jec­tion on Fri­day.

An ad­vi­so­ry com­mit­tee con­vened in May large­ly vot­ed against the la­bel ex­pan­sion, with some pan­elists cit­ing con­cerns about the pa­tient mix in the piv­otal STAR­G­LO study. About half of the pa­tients were in Asia — pri­mar­i­ly in Chi­na, Tai­wan and South Ko­rea — while on­ly 9% were in the US.

Sail Bio­med­i­cines, a well-fund­ed start­up that once as­pired to be­come Mod­er­na 2.0, is down­siz­ing as it tries to get its lead pro­gram — an in vi­vo CAR-T cell ther­a­py for au­toim­mune dis­ease — in­to the clin­ic, End­points News has learned.