FDA approves first oral blood thinning medication for children

FDAThe U.S. Food and Drug Administration approved Pradaxa (dabigatran etexilate) oral pellets to treat children 3 months to less than 12 years old with venous thromboembolism (a condition where blood clots form in the veins) directly after they have been treated with a blood thinner given by injection for at least five days. The FDA also approved Pradaxa oral pellets to prevent recurrent clots among patients 3 months to less than 12 years old who completed treatment for their first venous thromboembolism.

In addition, Pradaxa was approved in capsule form to treat blood clots in patients eight years and older with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least five days, and to prevent recurrent clots in patients eight years and older who completed treatment for their first venous thromboembolism.

Pradaxa is the first FDA-approved blood thinning medication that children can take by mouth; the only other approved blood thinning medication for children is given by injection. Pradaxa was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.

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

Blood clots can be a serious problem in children as well as adults. Children are most at risk for blood clots if they have cancer, congenital heart disease, a central venous catheter, or are admitted to an intensive care unit. Venous thromboembolism can lead to complications, including swelling and discomfort near the clot, chest pain, lung damage, and even death.

The safety and efficacy of Pradaxa for treating blood clots in patients younger than 18 was evaluated in one study of 267 pediatric patients. In this open-label study, patients were randomly assigned to receive either Pradaxa or standard of care. The study compared the two groups for the number of patients who met the composite endpoint (a combination of multiple clinical endpoints), which meant that they had not died from a blood clot, their blood clots had completely resolved, and they had no additional blood clots. Results showed that 81 (45.8%) of the 177 people taking Pradaxa met the composite endpoint compared to 38 (42.2%) of the 90 patients who received standard of care.

The safety of Pradaxa to prevent recurrent blood clots in the same pediatric population was evaluated in an open-label, single-arm study in 214 patients with a history of blood clots. The primary endpoints of the study were recurrence of blood clots, major and minor bleeding events, and death (both overall and related to blood clots). The safety of Pradaxa with long-term use was similar to the previously discussed study. Recurrence of blood clots occurred in three patients (1.4%), which was comparable to prior standard-of-care treatments.

The most common side effects of Pradaxa include digestive system symptoms and bleeding. Pradaxa can cause serious and fatal bleeding. Pradaxa is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome. Pradaxa 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 (spinal or epidural hematomas) in patients undergoing spinal procedures may cause serious side effects.

Pradaxa received a priority review designation for this indication.

The FDA granted the approval of Pradaxa to Boehringer Ingelheim Pharmaceuticals, Inc.

Most Popular Now

Pfizer and BioNTech receive positive CHMP opinion …

Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) announced a booster dose of their Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine (COMIRNATY® Original/Omicron ...

Vividion Therapeutics names Jenna Goldberg as Chie…

Vividion Therapeutics, Inc., a biopharmaceutical company utilizing novel discovery technologies to unlock high value, traditionally undruggable targets with precision the...

Bayer with continued strong performance

The Bayer Group maintained its strong business performance across all three divisions in the third quarter. "Despite rising inflation and global supply chain problems, we...

Sanofi and GSK's next-generation COVID-19 booster …

After the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for VidPrevtyn® Beta, the vaccine was approved by t...

GSK announces positive Phase IIa study results for…

GSK plc (LSE/NYSE: GSK) announced positive results from a Phase IIa study demonstrating that GSK3036656, a first-in-class investigational antitubercular agent, was well t...

Sugar molecules as a target in cancer therapy

Cancer cells use sugar molecules on their surface to disable attacks by the body's immune system. Researchers at the University of Basel now report on how this mechanism ...

COVID vaccination improves effectiveness of cancer…

Patients with nasopharyngeal cancer are often treated with drugs that activate their immune system against the tumor. Until now, it was feared that vaccination against Co...

Making melanoma immortal: Pitt scientists discover…

Scientists at the University of Pittsburgh School of Medicine have discovered the missing puzzle piece in the mystery of how melanoma tumors control their mortality. I...

Unusual type of antibody shows ultrapotent activit…

An unusual type of antibody that even at miniscule levels neutralizes the Zika virus and renders the virus infection undetectable in preclinical models has been identifie...

New drug shows promise for fighting both COVID-19 …

While vaccination can provide life-saving protection against COVID-19, scientists are still searching for ways to treat severe infections, including in people who cannot ...

Machine learning can help predict patient response…

Predicting which patients will respond well to treatment is a quandary that has plagued the field of cancer immunotherapy for more than four decades. Now, researchers at ...

Study reveals vaccine confidence declined consider…

A new study suggests that, despite the success of the COVID-19 vaccination campaigns, vaccine confidence has declined significantly since the start of the pandemic. Re...