Takeda Demonstrates Ongoing Commitment in Rare Hematology By Continuing to Gather Real-World Evidence and Advance Personalized Treatment

Takeda Pharmaceutical Company Limited  announced that 9 hematology abstracts are being presented at the 13th Annual Congress of the European Association for Haemophilia and Allied Disorders, EAHAD 2020. The data being presented demonstrate Takeda’s ongoing commitment in rare hematology by continuing to gather important real-world evidence, beyond its product portfolio, and advance personalized treatment for those living with bleeding disorders.

Gathering Real-World Evidence and Identifying Unmet Needs in Bleeding Disorders
The real-world evidence being presented by Takeda goes beyond its product portfolio and shines a spotlight on the important unmet needs faced by the wider bleeding disorders community. Specifically, literature review findings in the abstract “Incidence and Prevalence of Diagnosed and Undiagnosed Hemophilia A and Hemophilia B in the United Kingdom, Germany, France, Italy, and Spain” (abstract P050) reveal that the estimated percentage of undiagnosed cases in EU5 countries ranged from 16–47% for hemophilia A and 34–65% for hemophilia B. The diagnosis rates varied with disease severity, and the highest rates were typically observed for severe disease.

“Many of us are aware that hemophilia is underdiagnosed, but we don’t necessarily have access to data that show a comprehensive picture of the overall rate of undiagnosed cases in a certain region,” says Dan Curran, M.D., Head of the Rare Diseases Therapeutic Area Unit, Takeda. “Our study, which looks at the incidence and prevalence of diagnosed and undiagnosed Hemophilia A and Hemophilia B in EU5 countries, provides more clarity, and the findings continue to motivate our work with the bleeding disorders community to improve diagnosis.”

Takeda is also working tirelessly to better understand the efficacy and safety of its portfolio in a real-world setting, by continuously collecting data from clinics as well as patients who are being treated with its products. Some of the insights being presented at EAHAD 2020 include:

• VEYVONDI® [Recombinant von Willebrand factor (rVWF), vonicog alfa]: Menorrhagia is the most common symptom affecting females with von Willebrand disease (VWD). The abstract “Management of Menorrhagia in a Phase 3, Post-Hoc, Open-Label Study of rVWF in patients with severe VWD” (abstract P218) shows the hemostatic efficacy rating of vWVF in patients being treated with it.

• ADVATE® [Recombinant Antihemophilic Factor (rAHF), octocog alfa]: The abstract “AHEAD International and German studies: 6-year interim effectiveness and safety outcomes in patients with hemophilia A receiving antihemophilic factor (recombinant) in a real-world setting” (abstract P115) focuses on the long-term effectiveness and tolerability of rAHF, regardless of disease severity, in two study populations. AHEAD is one of the largest long-term real-world studies on hemophilia, conducted in 22 countries around the world.

• ADYNOVATE® [Recombinant Antihemophilic Factor, PEGylated, TAK-660]: In the abstract “Immunogenicity Profile of Rurioctocog Alfa Pegol in Previously Treated Patients with Severe Congenital Hemophilia A: Findings from 6 Clinical Trials” (abstract P187), researchers analyzed the association of PEGylated TAK-660 and the risk of developing FVIII inhibitors and persistent antibody response.

• FEIBA® [Anti-Inhibitor Coagulant Complex]: The abstract “Safety and Effectiveness of Activated Prothrombin Complex Concentrate (aPCC) Monotherapy in Patients with Hemophilia and Inhibitors (PwHI) Undergoing Surgery: A Systematic Review and Meta-Analysis” (abstract P114) shows the rate of adverse events in patients treated with aPCC monotherapy in a surgical setting, as well as its hemostatic effectiveness in different surgical procedures.

“Getting a treatment to market is just the beginning,” Curran adds. “We want to ensure our treatments benefit patients and their care team in the real world. Gathering evidence on an ongoing basis from day-to-day medical practice, outside of rigorous clinical studies, is critical. These data will continue to help define our R&D strategies and clinical trial designs.”

Advancing Personalized Treatment in Rare Hematology
In addition to a suite of real-world evidence, Takeda is showcasing its commitment to advancing individualized treatment in hematology, by highlighting the importance of tailoring dosing regimen of hemophilia treatment based on patients’ different pharmacokinetic (PK) profiles.

In the abstract “Rurioctocog Alfa Pegol PK-guided prophylaxis targeting two FVIII Trough Levels in Severe Hemophilia A Patients (PROPEL Phase 3 Study): Impact of Patient FVIII Half-Life on Consumption and Efficacy Outcomes” (abstract OR09), researchers carried out a post hoc analysis to evaluate the relationship between patient FVIII half-life and efficacy and consumption of prophylactic rurioctocog alfa pegol (TAK-660). The abstract describes the impact of FVIII half-life on the amount and frequency of TAK-660 doses needed to achieve target FVIII troughs.

“Healthcare is changing, and patients are expecting more from their care team,” comments Dr. med. Wolfhard Erdlenbruch, M.D., Vice President Head of Global Medical Affairs Hematology, Takeda. “As technology advances, we are able to tailor everything around us to suit our needs and that should include medical treatments. We cannot assume that one regimen or one dosing strategy is going to be suitable for all patients – that’s because although these patients may be living with the same condition, the variation in their individual pharmacokinetics, for example, may mean they respond to the same treatment differently. With this in mind, Takeda is always finding new ways to make sure treatments for our patients are as tailored to their individual needs as possible.”

Read the full press release, here.