Hemlibra Helps Keep Bleeding Under Control in Babies in HAVEN 7

Interim results support benefit of starting Hemlibra from birth for severe hem A

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A.

The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment.

That’s according to interim results from HAVEN 7 (NCT04431726), a Phase 3 clinical study testing the benefit of preventive treatment with Hemlibra in infants whose ages ranged from newborn to 1 year.

“These initial results support the benefit of starting Hemlibra from birth given that early preventative treatment is essential in infants,” Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, said in a press release. Roche owns Genentech, the company that markets Hemlibra.

The results were shared at last month’s American Society of Hematology annual meeting, which was held in New Orleans, Louisiana, and virtually, Dec. 10–13. The presentation was titled “Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study.”

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HAVEN 7 testing Hemlibra in babies with severe hem A and no FVIII inhibitors

Hemophilia occurs when the body does not make enough of the clotting factors that help the blood to clot and prevent excessive bleeding.

Most people with hemophilia need preventive treatment that involves regular injections of the clotting factors they are missing. But for young children, this may be too much of a burden, and preventive treatment may not be started until after their first year of life.

“Haemophilia can substantially reduce quality of life for those affected, starting at infancy, which is especially distressing for parents and caregivers,” Garraway said. “We continue to explore Hemlibra’s potential benefits to a broad range of people with haemophilia A.”

Hemlibra is a medication that’s used to prevent or reduce bleeding in adults and children with hemophilia A. It is designed to do the work that factor VIII (FVIII) — the clotting factor that people with the disease are unable to produce — normally does.

HAVEN 7 is testing how safe Hemlibra is and how well it works in babies with severe hemophilia A who haven’t developed inhibitors (neutralizing antibodies) to FVIII. It’s also testing its pharmacokinetics (how it moves into, through, and out of the body) and pharmacodynamics (its effects on the body).

These initial results support the benefit of starting Hemlibra from birth given that early preventative treatment is essential in infants

An interim analysis looked at 54 babies who had received at least one dose of Hemlibra for a median period of 42.1 weeks. Their median age was 4.5 months, and 24 (44.4%) were younger than 3 months. There were 24 (44.4%) untreated and 30 (55.6%) minimally treated babies.

There were a total of 77 bleeds in 31 (57.4%) babies. Most (88.3%) of these bleeds resulted from some sort of trauma; 14 of these bleeds were treated. A smaller proportion were spontaneous (6.4%) or resulted from a medical procedure (5.2%).

The annualized bleeding rate, a measure of the number of bleeds divided by the number of months in the reporting time window and multiplied by 12 (the number of months in a year), was 0.4 for all treated bleeds and 0.1 for treated joint bleeds.

Nearly half (42.6%) of the babies had zero bleeds, and 42 (77.8%) had zero treated bleeds.

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Nine (16.7%) babies experienced a side effect — a reaction at the injection site — related to Hemlibra. There were no intracranial hemorrhages (brain bleeds), and no new safety signals were reported.

“The results of this interim analysis indicate the efficacy and confirm the safety” of Hemlibra in babies with severe hemophilia A, the researchers wrote.

The primary analysis will be conducted at 52 weeks (one year), after which babies will continue to be monitored in the long run for up to seven years.