Boehringer Ingelheim receives positive CHMP opinion for use of Pradaxa® (dabigatran etexilate) for the treatment and prevention of venous thromboembolic events in children from birth to less than 18 years of age
- No approved therapy for the treatment and prevention of venous thromboembolic events in children until now
- Current standard of care often associated with frequent monitoring of anticoagulation or daily injections1
- Positive opinion is based on a dedicated clinical pediatric program for dabigatran´s, including the DIVERSITY study,2 which supplements dabigatran´s established and well-documented safety and efficacy profile in adults3
Ingelheim, Germany, 16. November 2020 – Boehringer Ingelheim today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on the proposed indication for Pradaxa® (dabigatran etexilate) for the treatment of venous thromboembolic events (VTE) and prevention of recurrent VTE in pediatric patients from birth to less than 18 years of age.
If the proposed indication is approved by the European Commission (EC), pediatric patients and healthcare professionals will have access to an oral anticoagulant therapy for which the risk-benefit profile has been thoroughly assessed by a health authority. At present, there is no approved therapy for the treatment or prevention of blood clots in veins (VTE) for children, and current standard of care (SOC) is associated with a range of limitations – including the need for frequent monitoring of anticoagulation level or burden of daily injections.1
“It is important that healthcare professionals have access to an oral anticoagulant treatment that has gone through rigorous testing, and is as convenient as possible for children,” said Leonardo R. Brandão, Thrombosis Program Director, Associate Professor, The Hospital for Sick Children, Department of Pediatrics, University of Toronto, Toronto, Canada. “If adopted by the EC, this positive opinion could potentially lead to the availability of a treatment option that can be administered orally, does not require routine laboratory monitoring or dose titration, and is available in formulations targeted to specific age groups, including younger patients.”
“Dabigatran has a proven positive safety profile in adults, demonstrated across clinical and real-world studies,3 and we are delighted that it has now been confirmed in pediatric patients too,” said Waheed Jamal, Corporate Vice President, Head of CardioMetabolism Respiratory Medicine, Boehringer Ingelheim. “This positive opinion provides further evidence that dabigatran is suitable for use across a wide range of patient populations, with clinical experience that equates to over 10 million patient-years across all licensed indications to date. If approved, it will bring a new therapy where there are currently no licensed treatment options available.”
The positive CHMP opinion is based on a dedicated pediatric clinical program. The DIVERSITY trial demonstrated that dabigatran was non-inferior to SOC for pediatric patients at high risk of VTE, with comparable bleeding rates,2 while the Brandão L et al. study showed favorable safety results with dabigatran in children with VTE and persistent thrombosis risk factors.1
The expected approval represents an important milestone for dabigatran, building on the outcomes of its associated trials and potentially widening its patient population.
About the DIVERSITY study, Albisetti M et al.
This open-label, randomized (1:2), parallel-group, phase 2b/3 non-inferiority trial evaluated the efficacy and safety of SOC versus dabigatran using a pediatric dabigatran dosing algorithm (age- and weight-adjusted nomogram) in children with acute VTE aged 12 to <18, 2 to <12, and birth to <2 years requiring anticoagulation therapy for ≥3 months.2
The primary composite efficacy endpoint was the proportion of children with complete thrombus resolution, and freedom from recurrent VTE or VTE-related death. The secondary endpoints were safety and pharmacokinetic/pharmacodynamic relationships.2
The results from the DIVERSITY trial demonstrated that dabigatran was non-inferior to SOC in efficacy for acute VTE treatment, with similar pharmacokinetic/pharmacodynamic relationships to adults, and may be a suitable alternative to SOC.2
About the secondary VTE prevention study, Brandão L et al.
The open-label, single-arm, prospective cohort, Phase III trial is the first study of its kind to describe outcomes in children treated with a direct oral anticoagulant for secondary VTE prevention. In the study, approximately 200 children received dabigatran for up to 12 months. The primary endpoints for this study included VTE recurrence, bleeding events and mortality at 6 and 12 months.1
The study showed a low overall frequency of recurrent VTEs and any bleeding events.1 Based on these results, the authors concluded that this trial showed favorable safety results with dabigatran in children with VTE and persistent thrombosis risk factors.1
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References
1 Brandão L. et al. Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children. Blood. 2020;135(7):491–504.
2 Updated DIVERSITY manuscript – publication pending: Albisetti et al. Dabigatran etexilate for the treatment of acute venous thromboembolism in children: results of an open-label, phase 2b/3, randomised clinical trial.
3 Boehringer Ingelheim, Data on File.