‘Hemlibra is the most advanced hemophilia A drug availble'

[Interview] Dr. Midori Shima, Professor of Pediatric Hematology/Oncology, Nara Medical University, Japan

Hemlibra used for prophylaxis maintains and improves joint health in pediatric patients...enabling patients to conduct daily sports activities

Confirmed not only the drug's therapeutic safety but also its effect in preventing bleeding in surgical patients

The only hemophilia A treatment for patients with and without antibodies who are resistant to factor VIII agents

Dr.

 

Midori Shima, Professor at Nara Medical University is explaining the therapeutic benefits of Hemlibra

"Hemlibra has the potential to become the standard of care for hemophilia A, and is considered the most advanced treatment available." Dr.

 

Midori Shima, professor of pediatric hematology-oncology at Nara Medical University in Japan, said so at a media session to mark the 1st anniversary of Hemlibra's insurance reimbursement extension at the InterContinental Seoul COEX.

 

In the Phase III HOHOEMI trial, pediatric participants under the age of 12 saw significant joint health benefits and improved quality of life after taking Hemlibra.

 

Professor Shima said, "Most people with hemophilia undergo knee and hip surgery in their 30s and 40s due to bleeding.

 

Although long-term follow-up is required, our current results suggest that starting Hemlibra in childhood can help maintain knee joint health well into middle age.” Also, the 3-year interim analysis results of the AOZORA study showed that the annualized bleed rate (ABR)  reductions seen in the HOHOEMI study were maintained.

 

The mean ABR decreased from 0.4 (0.00-4.55) to 0.2 (0.00-4.04) before and after treatment with Hemlibra, respectively, and 40% of participants (12) had no bleeds.

 

The AOZORA study will continue to track patients' MRI data through Week 313 and is expected to show that Hemlibra is effective in maintaining and improving joint health in pediatric hemophilia A patients.

 

In addition, Hemlibra has 8 years of post-marketing surveillance data, including exposure data from 22,000 patients worldwide, demonstrating its safety and efficacy.

 

For example, the Canadian registry study has efficacy and safety data from 553 patients of various lifestyles and ages.

 

The study confirmed efficacy in preventing long-term bleeding, with 72.9% of patients with severe hemophilia A experiencing no bleeding.

 

In addition, patient-to-patient comparisons showed a reduction in mean ABR.

 

The mean ABR before treatment with Hemlibra was 1.22, and the mean ABR after treatment was 0.50.

 

These data demonstrate that Hemlibra can safely and effectively prevent bleeding over the long term in a diverse patient population.

 

Dr.

 

Seema explained that Hemlibra prophylaxis offers multiple benefits for each patient group by age and lifestyle..

 

First, in pediatric patients, all previous prophylaxis treatments to reduce bleeding have required 2-3 intravenous injections per week, but Hemlibra can be administered as a subcutaneous injection once a week, once every two weeks, up to once every four weeks.

 

This extended duration and convenience in administration means that patients can start prophylaxis at a younger age, which can have a significant impact on quality of life.

 

"By starting prophylaxis early and maintaining regular prophylaxis, pediatric patients can maintain healthy joints without bleeding problems and prevent or minimize the impact of future arthropathies," said Dr.

 

Shima.

 

Hemlibra may also improve the patient’s ability to participate in academic and professional activities by reducing bleeding rates.

 

The bruising and scarring of blood vessels that can occur with intravenous infusions may be eliminated by switching to subcutaneous infusions, improving the quality of life during this important developmental stage of a patient's life, including the ability to form social relationships.

 

It is also notable that the risk of bleeding complications is significantly reduced in patients receiving Hemlibra, which may improve surgical outcomes.

 

Most minor surgeries (e.g., dental procedures) can be performed without the need for additional clotting factor infusions.

 

Therefore, the use of Hemlibra may minimize the need for transfusions of clotting factor concentrates or other blood products during and after surgery.

 

This means that potential complications associated with transfusions can be avoided, contributing to a faster recovery period and shorter hospital stay, which would allow patients to return to their daily activities sooner.

 

Meanwhile, JW Pharmaceutical's Hemlibra is an innovative new drug that works by mimicking the function of Factor VIII, a blood clotting factor that is deficient in the body of hemophilia A patients.

 

Bispecific antibody technology was applied to the drug to simultaneously bind to factor IXa and factor X.

 

Hemlibra is the only hemophilia A treatment that can be used in patients with and without antibodies who are resistant to existing factor VIII agents.

 

It provides long-lasting bleed prevention with subcutaneous injections once every up to four weeks.