He­mo­phil­ia groups warned of 5 deaths among pa­tients tak­ing Roche’s big new block­buster Hem­li­bra

The He­mo­phil­ia Fed­er­a­tion of Amer­i­ca has put out a state­ment not­ing that Roche alert­ed them of the deaths of 5 pa­tients who were tak­ing their new drug Hem­li­bra, cal­cu­lat­ed to be the biggest sin­gle drug launch of the year. And ac­cord­ing to Roche, the alert fol­lowed the death of two pa­tients ear­li­er this year, which they just learned about.

Ac­cord­ing to the state­ment, Roche’s Genen­tech in­formed them of the deaths Mon­day — adding 4 fa­tal­i­ties to the 1 death al­ready re­port­ed ear­ly last year.

The news trig­gered a 1.4% drop in Roche shares Wednes­day morn­ing.

Ac­cord­ing to the HFA:

Genen­tech has lit­tle in­for­ma­tion that they can present­ly share about the cir­cum­stances sur­round­ing the most re­cent pa­tient deaths; how­ev­er, Genen­tech could con­firm that the pa­tients had re­ceived Hem­li­bra as part of com­pas­sion­ate use and ex­pand­ed pa­tient ac­cess.

In a state­ment to End­points News late Tues­day, a spokesper­son for the com­pa­ny said that one of the pa­tients who died was in an ex­pand­ed ac­cess pro­to­col, three re­ceived it fol­low­ing com­pas­sion­ate use re­quests and one was in the HAVEN I tri­al. She added:

To date, five adults tak­ing Hem­li­bra have passed away. For all, the treat­ing physi­cian or in­ves­ti­ga­tor’s as­sess­ment was that the cause of death was un­re­lat­ed to Hem­li­bra (em­pha­sis pro­vid­ed by Roche). We pro­vid­ed the na­tion­al ad­vo­ca­cy groups with this up­date via phone yes­ter­day.

In a fol­lowup, Roche added a time­line:

  • In 2016, one adult tak­ing Hem­li­bra through a re­quest for com­pas­sion­ate use passed away.
  • In 2017, one adult par­tic­i­pat­ing in the Phase III HAVEN 1 clin­i­cal tri­al passed away and one adult tak­ing Hem­li­bra through a re­quest for com­pas­sion­ate use passed away.
  • In 2018, we just re­cent­ly learned that two adults tak­ing Hem­li­bra passed away. Again, the treat­ing physi­cian’s as­sess­ment for both pa­tients was that the cause of death was un­re­lat­ed to Hem­li­bra.

Roche $RHH­BY has had safe­ty is­sues to deal with be­fore on this drug.

The phar­ma gi­ant re­port­ed 2 cas­es of throm­boem­bol­ic events and 3 in­stances of throm­bot­ic mi­croan­giopa­thy from the Haven I Phase III tri­al. Roche rat­tled its most bull­ish fol­low­ers on this drug with the re­port that a pa­tient died on Feb­ru­ary 22, 2017 from a rec­tal he­m­or­rhage af­ter be­ing treat­ed with emi­cizum­ab and a by­pass­ing agent dur­ing a break­through bleed­ing episode. The in­ci­dents were at­trib­uted to the BPAs, which an­gered Shire and No­vo Nordisk, the two key ri­vals who are feel­ing the biggest im­pact from the ar­rival of Hem­li­bra.

Shire in par­tic­u­lar came out gun­ning against Hem­li­bra, which has a peak sales es­ti­mate of $4 bil­lion. Shire has claimed be­fore that Roche has is­sued “in­ac­cu­rate and mis­lead­ing” state­ments about the se­ri­ous ad­verse events tracked in its Phase III – events which con­spired to keep this would-be block­buster un­der a cloud.

Roche, though, nev­er blinked, bag­ging an ap­proval in the US No­vem­ber 17 for one of its biggest drugs in the pipeline, a key win that was bad­ly need­ed to ac­count for the loss of patent pro­tec­tion on 3 big fran­chise drugs. The EU fol­lowed with an OK Feb­ru­ary 18 and Japan fol­lowed suit March 18.

In the US, the FDA added a boxed warn­ing about safe­ty haz­ards. When the drug was ap­proved last fall, the agency not­ed:

Cas­es of throm­bot­ic mi­croan­giopa­thy and throm­bot­ic events were re­port­ed when on av­er­age a cu­mu­la­tive amount of >100 U/kg/24 hours of ac­ti­vat­ed pro­throm­bin com­plex con­cen­trate (aPCC) was ad­min­is­tered for 24 hours or more to pa­tients re­ceiv­ing emi­cizum­ab-kxwh pro­phy­lax­is. The pre­scrib­ing in­for­ma­tion con­tains a boxed warn­ing to mon­i­tor for throm­bot­ic mi­croan­giopa­thy and throm­bot­ic events when aPCC is ad­min­is­tered. If symp­toms oc­cur, aPCC should be dis­con­tin­ued and emi­cizum­ab-kxwh should be sus­pend­ed.

The HFA added:

Genen­tech has a Med­ical Com­mu­ni­ca­tions line at 1(800)-821-8590 for pa­tients, con­cerned com­mu­ni­ty mem­bers, and health­care providers who seek fur­ther in­for­ma­tion. HFA en­cour­ages you to reach out to Genen­tech and/or to your treat­ing physi­cian if you have ques­tions or con­cerns.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).