Sarep­ta rock­ets up af­ter FDA ap­proves its con­tro­ver­sial Duchenne drug

Af­ter years of fierce de­bate in a see­saw bat­tle over the fate of Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy drug eteplirsen, the FDA has grant­ed the drug con­di­tion­al ap­proval in the US, mak­ing this the first drug OK’d to treat the lethal dis­ease.

Janet Wood­cock

The ap­proval comes with the threat of swift ret­ri­bu­tion, though, if in­ves­ti­ga­tors fail to fol­low through with pos­i­tive da­ta from a big­ger study. The agency’s state­ment in­cludes a re­quire­ment that Sarep­ta will now have to con­firm that the drug ac­tu­al­ly works as de­signed, spurring pro­duc­tion of dy­s­trophin in a way that can im­prove mo­tor func­tion in the 13% of pa­tients amenable to ex­on 51 skip­ping. And the agency says that it may with­draw its OK with­out that da­ta.

For now, though, Sarep­ta will move fast to start mar­ket­ing the drug.

The agency’s de­ci­sion came months af­ter a pan­el of out­side ex­perts vot­ed against an ap­proval and FDA in­sid­ers, no­tably Ron Farkas, who re­cent­ly left the agency, re­ject­ed the biotech’s ar­gu­ment at every turn this year. Par­ents and pa­tients, though, turned out in the hun­dreds to voice their full sup­port for eteplirsen, mount­ing a lob­by­ing cam­paign that would re­cruit a long list of elect­ed of­fi­cials as well as physi­cians who treat this dis­ease.

Sarep­ta’s $SRPT share price, which has been on a roller coast­er ride at every twist and turn in the eteplirsen saga, ripped up 81% on the news. The drug will now be sold as Ex­ondys 51.

The drug, though, will re­main con­tro­ver­sial. The agency’s Janet Wood­cock over­ruled oth­ers in the FDA in giv­ing this drug a green light, in­clud­ing El­lis Unger, the di­rec­tor of the Of­fice of Drug Eval­u­a­tion, who protest­ed against Wood­cock’s de­ci­sion. And while the drug has its avowed sup­port­ers, a le­gion of crit­ics has not­ed that Sarep­ta nev­er pro­vid­ed the da­ta need­ed to win an ap­proval, with Wood­cock seen as cav­ing to the lob­by­ing cam­paign in a way that would like­ly dog the agency for years to come.

The big de­bate over this drug cen­tered on its tiny pi­lot study, which gath­ered da­ta on on­ly a dozen boys. Sarep­ta says that their in­ves­ti­ga­tors gath­ered clear ev­i­dence that their drug helped boys with this crip­pling, dev­as­tat­ing dis­ease con­tin­ue to walk fur­ther dis­tances than his­tor­i­cal da­ta would con­sid­er pos­si­ble with­out a ther­a­peu­tic ef­fect. FDA in­sid­ers like Farkas, though, re­ject­ed the his­tor­i­cal com­par­isons and crit­i­cized the biotech for fail­ing to pur­sue a larg­er study ear­li­er.

The ap­proval comes af­ter sev­er­al po­ten­tial ri­vals have been slapped down, with Bio­Marin’s can­di­date re­ject­ed — and now shelved — and PTC’s drug re­fused a hear­ing af­ter in­sid­ers at the agency con­clud­ed that the com­pa­ny sim­ply had not made its case.

Those ear­li­er re­fusals leave Sarep­ta with a clear shot at its mar­ket.

Janet Wood­cock, the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search di­rec­tor, said:

“Pa­tients with a par­tic­u­lar type of Duchenne mus­cu­lar dy­s­tro­phy will now have ac­cess to an ap­proved treat­ment for this rare and dev­as­tat­ing dis­ease. In rare dis­eases, new drug de­vel­op­ment is es­pe­cial­ly chal­leng­ing due to the small num­bers of peo­ple af­fect­ed by each dis­ease and the lack of med­ical un­der­stand­ing of many dis­or­ders. Ac­cel­er­at­ed ap­proval makes this drug avail­able to pa­tients based on ini­tial da­ta, but we ea­ger­ly await learn­ing more about the ef­fi­ca­cy of this drug through a con­fir­ma­to­ry clin­i­cal tri­al that the com­pa­ny must con­duct af­ter ap­proval.”

 

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

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