Drug Development

Faced with fresh concerns about Hemlibra’s safety, Roche spells out what it knows about 5 deaths

Roche is offering some additional details on the 5 deaths they’ve recorded among hemophilia patients taking Hemlibra, repeating their assertion that the risk/benefit profile of the would-be blockbuster remains unchanged as they conduct an “ongoing” investigation of two recent cases.

According to a spokesperson, 2 new deaths were recorded recently which physicians linked to a pre-existing condition in one case and major vascular surgery in another. Those cases followed deaths associated with a rectal hemorrhage and two intracranial hemorrhages.

News of the death tally, which spread like wild fire after Roche sent out a statement to patient groups, reignited lingering safety concerns about the drug, which analysts believe has been on track to rack up $4 billion to $5 billion in annual sales.

David Steinberg

Jefferies analyst David Steinberg highlighted the statement from Roche and some continued puzzlement over the drug’s mechanism of action, writing that these newly reported deaths could arouse fresh safety concerns. He noted:

It is Possible that Concerns Over Hemlibra’s Safety Could Now Reemerge Amongst Clinicians and Patients: Importantly, the NHA – which received information from Roche’s clinical development lead on March 26 – further indicated that these deaths have all apparently been deemed unrelated to Hemlibra by investigators or treating physicians. However, given the previously reported death and cases of thromboembolic events and thrombotic microangiopathy observed during clinical testing in a relatively small number of patients – attributed to the concurrent use of Hemlibra with bypassing agents (Feiba/Novo 7) during breakthrough bleeding episodes – it is not entirely surprising to see additional disclosures of serious additional adverse events, including unfortunately – deaths. And with very limited information, it is unclear if there are additional safety concerns with Hemlibra at this point. However, it’s a possibility that heightened concerns – which arose after the initial death in the HAVEN 1 study – could remerge. Additionally, we’d note that some experts still do not fully understand the mechanism behind Hemlibra, which could potentially impact use in non-inhibitor patients who are generally well controlled on SHPG’s Advate.

And for its part, Shire — facing a major rival to a key franchise program — has stoked fears as well, claiming earlier that Roche has made “inaccurate and misleading” statements about the serious adverse events tracked in its Phase III.

According to the spokesperson for Roche, here’s what they know about the deaths:

— In 2017, the adult patient in the Phase III HAVEN 1 trial experienced a serious rectal hemorrhage, which the investigator assessed as the cause of death, unrelated to Hemlibra.

— Three deaths have been reported to Genentech in adult patients taking Hemlibra through requests to Roche for compassionate use.

— In 2016, an adult patient taking Hemlibra through a request for compassionate use passed away due to an intracranial hemorrhage and widespread bacterial infection, which the treating physician assessed as the cause of death, unrelated to Hemlibra.

— In 2017, an adult patient taking Hemlibra through a request for compassionate use passed away. The patient had suffered multiple intracranial hemorrhages in the past due to predisposing factors, prior to starting Hemlibra. The treating physician’s assessment was that the cause of death was due to an intracranial hemorrhage, and it was unrelated to Hemlibra.

— We just recently learned that a third adult patient taking Hemlibra through a request for compassionate use passed away. The treating physician’s assessment was that the cause of death was due to a pre-existing medical condition, and it was unrelated to Hemlibra. It was reported that the patient had been enrolled in the compassionate use program due to a medical emergency related to an abdominal pseudo-tumor and passed away from complications related to this pre-existing condition.

— We just recently learned that a fifth adult patient taking Hemlibra passed away. It was reported that the patient passed away several weeks following an elective major vascular surgery. The treating physician’s assessment was that the patient’s death was unrelated to Hemlibra.

Our investigation and assessment of these two recent events is currently ongoing. We understand the urgency of reviewing and assessing safety events, and it is a priority for us to share accurate information about the safety of Hemlibra that is based on a thorough assessment. We will work diligently to provide appropriate updates as quickly as possible, while ensuring the confidentiality of the patients is respected. As mentioned previously, based on these assessments and the available information, these events do not change the currently known benefit/risk profile of Hemlibra.

We’ll keep you posted as any new details come in. It’s unlikely the safety spotlight will fade anytime soon.


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