Setbacks

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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BIO 2018

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