Trial findings back wider use of Roche’s Hemlibra

Currently, the therapy is approved to treat those with the condition who have factor VIII inhibitors, that is antibodies causing resistance to replacement clotting factors.

However, data from the Haven III and Haven IV trials clearly show Hemlibra’s efficacy in preventing bleeds in patients without inhibitors, in those who received prior therapy and in those who didn’t.

In the Phase III HAVEN 3 study, adults and adolescents aged 12 years or older without factor VIII inhibitors who received Hemlibra prophylaxis every week or every two weeks showed a 96 percent and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis.

In addition, 55.6 percent of people treated with Hemlibra every week and 60 percent of those given the drug every two weeks experienced zero treated bleeds, compared to 0 percent of people treated with no prophylaxis.

Also of note, in an intra-patient comparison, in patients who were previously enrolled in a prospective non-interventional study (NIS), once-weekly Hemlibra prophylaxis showed superior efficacy compared to prior factor VIII prophylaxis, as shown by a 68 percent reduction in treated bleeds.

"Hemlibra is the first medicine to show superior efficacy to prior factor VIII prophylaxis, the current standard of care therapy, as demonstrated by a statistically significant reduction in treated bleeds in the HAVEN 3 study intra-patient comparison," said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa.

"Even with current prophylactic treatments, many people with haemophilia A continue to have bleeds that can lead to long-term joint damage, and there is a need for more treatment options."

The drug also fared well on the safety side, with no unexpected or serious adverse events (AEs) reported, the most common being injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.

"With this data, we now have positive results from all four of our Phase III trials that reinforce the overall efficacy and safety of Hemlibra and its potential to improve care for all people with haemophilia A," said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development.

In the single-arm Phase III HAVEN 4 study, adults and adolescents with or without factor VIII inhibitors receiving Hemlibra prophylaxis every four weeks had a median annualised bleeding rate for treated bleeds of 0, with 56.1 percent experiencing zero treated bleeds and 90.2 percent experiencing three or fewer treated bleeds.

These results show that Hemlibra administration every four weeks "can provide clinically meaningful control of bleeding in people with haemophilia A with or without factor VIII inhibitors," the firm noted.

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