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.”

 

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Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

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The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

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Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

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That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

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