Dabigatran Etexilate (Pradaxa) Becomes First Oral Blood-Thinning Option for Use In Pediatric Patients

The US Food and Drug Admistration (FDA) has granted approval to dabigatran etexilate (Pradaxa), the first oral blood thinning medication for use in children in the organization’s history.

Announced in a statement from the FDA, the decision means dabigitran etexilate oral pellets are now indicated for use in children aged 3 months to less than 12 with venous thromboembolism (VTE) directly after treatment with injectable blood thinners for at least 5 days. The statement noted the FDA also approved dabigatran etexilate for prevention of recurrent clots for the same age group in patients who have already completed treatment for their first VTE.

“The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take,” said Ann Farrell, MD, Director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research, in the aforementioned statement. “With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”

With approval awarded to Boehringer Ingelheim Pharmaceuticals, Inc., dabigatran etexilate oral pellets became the first oral blood-thinning option for children to receive FDA approval. The approval for treatment of VTE was based on the results of the DIVERSITY trial while the approval for prevention of recurrent blood clots is based on data from a secondary VTE prevention study with an open-label, single-arm design.

DIVERSITY assessed the safety and efficacy of dabigatran etexilate oral pellets in a group of 267 patients aged 18 years and younger. In this phase 2b/3 trial, dabigatran etexilate was compared against standard of care in patients with acute VTE requiring anticoagulation therapy for 3 months. Using a composite endpoint of recurrent VTE, VTE-related death, and thrombus resolution as the primary outcome, results indicated the primary outcome occurred in 45.8% of patients receiving dabigatran etexilate and 42.2% of those receiving standard care, which investigators suggested demonstrated dabigatran etexilate was non-inferior to standard of care in pediatric patients.

The study used in the approval for preventing recurrent VTE was designed as an open-label, single-arm, prospective cohort, Phase III trial and, according to Boehringer Ingelheim, was the first study of its kind to describe outcomes in children treated with a DOAC for secondary VTE prevention. Conducted in a population of 214 pediatric patients with a history of VTE, the study had multiple primary endpoints, including recurrence of blood clots, bleeding events, and death. Results of this study demonstrated the recurrence of blood clots in the dabigatran etexilate was comparable to prior standard of care treatments and the long-term safety of dabigatran etexilate was similar to that observed in the DIVERSITY trial.

The FDA’s statement pointed out the most common side effects seen with use of dabigatran etexilate included digestive symptoms and bleeding. The statement also noted this medication is not recommended for those with bioprosthetic heart valves or triple-positive antiphospholipid syndrome. Additionally, the agent has a boxed warning cautioning that early treatment discontinuation may increase the risk of blood clots and that blood accumulation within parts of the spinal cord in patients undergoing spinal procedures may cause serious side effects.

This is a breaking news story and will be updated when new information becomes available.