Roche's Hemlibra (emicizumab-kxwh) has been approved by the FDA for adults and children with haemophilia A with factor VIII inhibitors and is awaiting a regulatory decision in the EU. We are snap-polling US and EU5 haematologists to gauge reaction to its labelling and potential utilisation.
In the Phase III HAVEN 1 study, patients 12 years and older who received Hemlibra prophylaxis had a reduction in treated bleeds of 87 percent compared to those who received no prophylaxis. Hemlibra prophylaxis also reduced treated bleeds by 79 percent compared to previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study (NIS) prior to enrolment. Interim results from the pivotal HAVEN 2 study in children younger than 12 years showed that 87 percent who received Hemlibra prophylaxis experienced zero treated bleeds.
How compelling are these efficacy data?
Labelling for Hemlibra includes a boxed warning with the following "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 HEMLIBRA prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of HEMLIBRA if symptoms occur."
To what extent will this warning (and assuming a very similar warning on EU labelling) impede utilisation of Hemlibra?
How comfortable would you be switching a typical haemophilia A with factor VIII inhibitors patient from their existing treatment to Hemlibra (note also: the most common adverse events occurring in 10 percent or more of people treated with Hemlibra in pooled studies were injection site reactions, headache and joint pain. Hemlibra can be self-administered once weekly by subcutaneous injection).
As a result of its boxed warning (and assuming EU labelling is similar) and based on your anticipated utilisation of Hemlibra, what effect will it have on your utilisation of aPCC/FEIBA?
No material impact
Marginal decline in use
Moderate decline in use
Significant decline in use
Very significant decline in use
Do you perceive Hemlibra to be practice changing for the treatment of haemophilia A with factor VIII inhibitors?
Yes – in the short term
Yes – in the medium term
Yes – in the long term
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