All revved up and ready to roll, Roche is hand­ed the keys to a multi­bil­lion-dol­lar he­mo­phil­ia mar­ket

Roche has a green light to great­ly ex­pand its sales ef­fort be­hind Hem­li­bra, the land­mark he­mo­phil­ia drug that is mak­ing mar­ket­ing life mis­er­able at Shire.

The FDA has rolled out an ap­proval for the glob­al heavy­weight to start sell­ing their drug — a break­through that has nev­er­the­less stirred some un­easi­ness on the safe­ty side — to pa­tients who have yet to de­vel­op fac­tor VI­II in­hibitors. That mar­ket is sev­er­al times larg­er than the ini­tial group Roche gained ac­cess to last year.

The news was wide­ly ex­pect­ed. Roche’s ef­fi­ca­cy da­ta — 96% and 97% re­duc­tion in bleeds based on treat­ments de­liv­ered ei­ther week­ly or every two weeks — had al­ready in­spired an­a­lysts to mark this drug down as a po­ten­tial $5 bil­lion a year block­buster. Roche has al­so been fine tun­ing its game plan for the mar­ket­ing team, which has roiled ri­vals at Shire and oth­er fac­tor VI­II play­ers.

That headache will soon be­long to Christophe We­ber, the CEO at Take­da. We­ber, though, had plen­ty of time to run the num­bers on the im­pact on the fran­chise, which they’ll con­trol once the Shire buy­out is com­plete.

Michael Callaghan

Hem­li­bra is one of the stars of a new cast of drug play­ers for Roche, which needs new mon­ey to re­place some of the old­er rev­enue streams from its ag­ing, un­pro­tect­ed main­stays like Avastin. Roche ex­ecs record­ed rev­enue of 23 mil­lion Swiss francs for the drug in Q1, say­ing they were pleased by the ear­ly trac­tion they were gain­ing.

The drug, though, has a po­ten­tial­ly dan­ger­ous side. Back in the spring Roche alert­ed pa­tient groups to a case in­volv­ing one per­son who de­vel­oped an an­ti-drug an­ti­body to Hem­li­bra, forc­ing them off ther­a­py. It’s not near­ly enough to pre­vent reg­u­la­tors from get­ting the break­through drug to pa­tients, but it does cre­ate risk that has to be fac­tored in­to the equa­tion.

“(W)e now have a new class of med­i­cine for the first time in near­ly 20 years,” said Michael Callaghan, hema­tol­o­gist, Chil­dren’s Hos­pi­tal of Michi­gan, De­troit. “Hem­li­bra can re­duce bleeds, and it of­fers a new sub­cu­ta­neous ad­min­is­tra­tion once week­ly, every two weeks or every four weeks.” 

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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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

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. 

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? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

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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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.

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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