Hemlibra Helps to Prevent Bleeds in Mild, Moderate Hem A, Data Show

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial.

Results will be presented at the annual meeting of the American Society of Hematology (ASH) in December in an oral presentation, titled “Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study.”

“Our data at ASH reinforce our conviction that following the science and developing versatile treatment approaches leads to improved outcomes for patients in increasingly meaningful ways,” Levi Garraway, MD, PhD, said in a press release. Garraway is the chief medical officer and head of global product development at Roche, which markets Hemlibra.

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Hemophilia A is caused by the lack of a clotting protein called factor VIII (FVIII). Hemlibra is an approved antibody-based therapy that is designed to prevent bleeding by mimicking the activity of the FVIII protein.

Prior clinical trial data indicate that Hemlibra is generally safe and effective at preventing bleeding episodes in people with hemophilia A. The therapy is administered by subcutaneous (under-the-skin) injection.

HAVEN 6 (NCT04158648) is a Phase 3 trial that Roche is sponsoring to further study the safety, effectiveness, and pharmacological properties of Hemlibra in patients with mild to moderate hemophilia A without FVIII inhibitors — neutralizing antibodies that may render standard hemophilia A replacement therapies useless.

Interim data from HAVEN 6 to be presented at ASH was based on information from 71 patients (69 men and two women, with a median age of 23 years) treated with Hemlibra. Most of them (84.5%) were white.

There were 20 (28.2%) participants with mild hemophilia (FVIII levels less than 40% of normal, but higher than 5%), and the remaining 51 (71.8%) had moderate hemophilia (FVIII levels of 1%–5% of normal). Just over half (52.1%) of participants had previously been on prophylactic (preventive) treatment for bleeds.

All participants were first given loading doses of Hemlibra at 3 mg/kg once per week for four weeks. Thereafter, they received maintenance doses of the medication: either 1.5 mg/kg once weekly, 3 mg/kg every two weeks, or 6 mg/kg every four weeks.

Patients were followed up for a median of 27.5 weeks, or just over half a year.

Of the 71 participants, 49 (69%) reported at least one adverse event (side effect). The majority of adverse events (84.5%) were deemed by trial investigators not to be related to Hemlibra. The most common adverse event was headache (14.1%), and 12.7% of participants experienced injection site reactions.

No serious adverse events related to Hemlibra were reported. Two participants developed antibodies against Hemlibra. However, these antibodies had no apparent impact on the therapy’s effectiveness or pharmacological properties.

Nearly half (46.5%) of the participants have so far been bleed-free, and more than four out of every five (80.3%) have had no bleeds that required treatment. Over 90% have had no joint bleeds that required treatment, and a marked improvement in joint health over the course of treatment was observed.

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Patient-reported data indicated that, over the course of the trial, participants were feeling less burdened by treatment than they had before. In a survey of 50 participants, all but two (96%) said they preferred Hemlibra over their prior medication.

“Data at the [interim analysis] of the HAVEN 6 study indicate that [Hemlibra] has a favorable safety profile and is efficacious in bleed prevention for persons with mild or moderate” hemophilia A, the researchers concluded, noting that these results are in line with prior clinical trial data on the therapy.