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.

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Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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