FDA turns thumbs down on Al­ler­gan's op­ti­mistic case for uter­ine fi­broid drug as liv­er dam­age threat tor­pe­does ap­pli­ca­tion

One of Al­ler­gan’s $AGN top late-stage drugs just ran in­to a brick wall at the FDA.

The com­pa­ny not­ed Tues­day evening that the agency turned thumbs down on its mar­ket­ing ap­pli­ca­tion to start sell­ing ulipristal ac­etate for the treat­ment of ab­nor­mal uter­ine bleed­ing in women with uter­ine fi­broids. The state­ment is a lit­tle hazy on specifics, but Al­ler­gan cit­ed safe­ty con­cerns raised out­side the US — where the drug is al­ready avail­able.

Al­ler­gan had been up­beat about its late-stage da­ta high­light­ing high­ly sta­tis­ti­cal­ly sig­nif­i­cant ef­fi­ca­cy da­ta. And it bad­ly need­ed a US ap­proval to get this drug to the $1 bil­lion block­buster po­ten­tial it out­lined for in­vestors.

The new bot­tom line:

The agency cit­ed safe­ty con­cerns re­gard­ing ES­MYA post-mar­ket­ing re­ports out­side the Unit­ed States. Al­ler­gan plans to meet with the FDA to dis­cuss their com­ments and next steps.

And that could take a con­sid­er­able amount of time to nail down, de­pend­ing on the de­tails of the CRL.

Al­ler­gan has been forced to rein in its mar­ket­ing on the drug — sold as Es­mya — in Eu­rope as reg­u­la­tors raised con­cerns about the risk of rare but se­ri­ous liv­er dam­age. The Eu­ro­pean group has re­strict­ed sales “fol­low­ing re­ports of se­ri­ous liv­er in­jury, in­clud­ing liv­er fail­ure lead­ing to trans­plan­ta­tion” which were linked to the drug.

All that, though, could add up to some­thing worse than a sin­gle re­jec­tion might im­ply. Al­ler­gan CEO Brent Saun­ders said last May that the com­pa­ny was look­ing to sell its women’s health unit in or­der to right a list­ing stock price. But with Es­mya un­der a dark cloud, it will be un­like­ly to fetch the price it needs to im­press share­hold­ers.

Ab­b­Vie, mean­while, is pur­su­ing an OK for elagolix for uter­ine fi­broids, which scored it’s first ap­proval just weeks ago for en­dometrio­sis.




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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Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources. 

As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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UP­DAT­ED: Pay­back? Sarep­ta stunned as FDA spurns fol­lowup to Ex­ondys 51 for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.