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.”

 

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.