FDA Approves Two New Indications for Xarelto to Help Prevent and Treat Blood Clots in Pediatric Patients – PharmaLive

December 23, 2021 by No Comments

FDA Approves Two New Indications for XARELTO® (rivaroxaban) to Help Prevent and Treat Blood Clots in Pediatric Patients
— XARELTO® is available as both an oral tablet and oral suspension formulation for use in appropriate children less than 18 years of age
— Convenient oral suspension formulation advances standard of care for children; alleviates administration challenges found with injectable alternatives
— XARELTO® now has 11 indications, the most of any direct oral anticoagulant (DOAC), and is the only Factor Xa anticoagulant to offer flexible weight-based dosing for pediatric patients
Janssen Pharmaceutical Companies of Johnson & Johnson
Dec 20, 2021, 23:19 ET
RARITAN, N.J., Dec. 20, 2021 /PRNewswire/ — The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has approved two pediatric indications for XARELTO®(rivaroxaban): the treatment of venous thromboembolism (VTE, or blood clots) and reduction in the risk of recurrent VTE in patients from birth to less than 18 years after at least five days of initial parenteral (injected or intravenous) anticoagulant treatment; and thromboprophylaxis (prevention of blood clots and blood-clot related events) in children aged two years and older with congenital heart disease who have undergone the Fontan procedure. XARELTO® is the only direct oral anticoagulant (DOAC) FDA-approved for primary prevention of clots in pediatric patients following the Fontan procedure and the only DOAC in the U.S. to offer an oral suspension formulation for flexible, body weight-adjusted dosing options for pediatric patients.
CLICK TO TWEET: #BREAKINGNEWS: @US_FDA approves @JanssenUS therapy for the treatment and prevention of #bloodclots in #pediatric patients, providing an alternative to injectable anticoagulants that have long been the standard of care. Learn more: https://bit.ly/3E9qAOk
While VTE more commonly occurs in adults, blood clots can still be a serious problem in children, affecting approximately 58 per 10,000 of those hospitalized in the U.S., with rates increasing.1 Children may be at greater risk of blood clots when suffering from other conditions, such as infectious diseases, active cancer, or after undergoing surgery, like the Fontan procedure, which is performed in children who have a single functioning heart ventricle to redirect blood flow from the lower body to the lungs.2,3
Current medical guidelines are limited and recommend that young patients with or at risk for developing blood clots be treated with standard anticoagulation therapy, such as warfarin or heparin.4 Often times, physicians have to adjust adult doses, based on limited data for these therapies for younger patients.4 Additionally, for some of these treatment options, that can mean painful injections, dietary restrictions and regular laboratory monitoring – all things that can be especially challenging for younger patients and their caregivers.4,5,6
“Historically, there has been limited guidance and options for healthcare providers on how to help reduce potentially serious, even fatal, blood clots and related events in young children,” said Andrew Van Bergen, M.D., Pediatric Cardiologist, Advocate Children’s Hospital.* “We have had to adjust adult doses of standard anticoagulation therapies, which are both burdensome and uncomfortable for patients, and require frequent monitoring. Now that XARELTO® is FDA-approved with weight-based dosing options, either as tablets or liquid formulation, a convenient option is available allowing flexibility to tailor the treatment for my patients. This is a major advancement in antithrombotic care for those patients under the age of 18.”
The oral suspension formulation will be administered through a color-coded dosing device that was designed to help minimize dosing errors and is expected to become available in the U.S. for pediatric patients in mid-January 2022. The oral tablets are currently available in the U.S. for appropriate pediatric patients.
“When a child is experiencing health challenges, learning that they are also at risk for a blood clot can feel overwhelming for the patient, and also their parents or caregivers,” said Andrea Baer, MS, BCPA, Executive Director of The Mended Hearts, Inc., a patient advocacy organization whose program Mended Little Hearts serves patients and families with congenital heart disease.** “Knowing that there is now an FDA-approved oral treatment option to reduce the risk of blood clots that’s easy and may be more comfortable than injections to administer may help ease that burden.”
CLICK TO TWEET: #DYK Certain children are at risk for potential #bloodclots? Learn more about a new treatment option from @JanssenUS that provides weight-based dosing: https://bit.ly/3E9qAOk
XARELTO® now has 11 indications in the U.S. – the most of any DOAC – and is the most studied oral Factor Xa inhibitor in its class. This latest approval is based on two Phase 3 pediatric studies from the industry-leading EXPLORER clinical research program, EINSTEIN-Jr, the largest study to date evaluating pediatric patients from birth to <18 years of age with previously diagnosed VTE; and UNIVERSE, the first clinical trial to examine a DOAC for the prevention of VTE in pediatric patients after recently undergoing the Fontan procedure.7,8
“Today’s FDA approval marks two new XARELTO® indications for pediatric patients, an often underrecognized, but especially important patient population,” said James List, M.D., Ph.D., Global Therapeutic Area Head, Cardiovascular, Metabolism, & Retina, Janssen Research & Development, LLC. “At Janssen, we are committed to addressing unmet medical needs and the approval of the 10th and 11th indications for XARELTO® underscores its capability in reducing the risk of blood clots and cardiovascular events in patients from young to old and with a variety of conditions.”
In 2021, Janssen’s development partner, Bayer, received approval for XARELTO® in Canada, the European Union, the United Kingdom, Japan, Switzerland and in various Latin American countries for the treatment of VTE and prevention of VTE recurrence in the pediatric population, from birth to adolescents less than 18 years after at least five days of initial parenteral anticoagulation treatment.
EINSTEIN-Jr was a randomized, multicenter, active-controlled, open-label Phase 3 study that evaluated the use of XARELTO® in 500 children, aged birth to 17 years, with previously diagnosed acute VTE who had started parenteral anticoagulation therapy. Participants were enrolled from November 2014 to September 2018, from 107 sites in 28 countries, and were assigned in a 2:1 ratio to receive either an open-label, body weight-adjusted dose of XARELTO® to approximate a 20-mg adult dose (tablets or a new oral suspension) (n=335) or standard anticoagulation therapy (n=165). EINSTEIN-Jr is the largest pediatric study completed to date in the entire pediatric age population for the treatment of VTE. It is part of the comprehensive EINSTEIN program, which also included four pivotal Phase 3 studies in adult populations: EINSTEIN-DVT, EINSTEIN-PE, EINSTEIN-EXT and EINSTEIN CHOICE.
UNIVERSE was a randomized, multicenter, open-label, active-controlled, two-part, Phase 3 study that examined the use of a novel, oral suspension XARELTO® formulation in children 2-8 years old with single ventricle physiology who had the Fontan procedure within four months before enrollment. From November 2016 to June 2019, a total of 112 participants were enrolled across 36 sites in 10 countries.
Part A evaluated the single- and multiple-dose pharmacokinetic (PK) and pharmacodynamic (PD) properties of XARELTO® while Part B evaluated the comparative safety and efficacy of XARELTO® versus aspirin when used for thromboprophylaxis for 12 months.
A collaborative effort between Bayer and Janssen, our industry-leading EXPLORER program seeks to generate important clinical evidence on the safety and efficacy of XARELTO® and its potential role in addressing a wide range of critical medical needs. EXPLORER is unmatched by any oral anticoagulant in the Factor Xa inhibitor class in its size, scope and ambition.
WHAT IS XARELTO® (rivaroxaban)?
XARELTO® is a prescription medicine used to:
XARELTO® is used with low dose aspirin to:
XARELTO® is used in children to:
XARELTO® was not studied and is not recommended in children less than 6 months of age who:
XARELTO® may cause serious side effects, including:
Do not stop taking XARELTO® without talking to the doctor who prescribes it for you. Stopping XARELTO® increases your risk of having a stroke. If you have to stop taking XARELTO®, your doctor may prescribe another blood thinner medicine to prevent a blood clot from forming.
You may have a higher risk of bleeding if you take XARELTO® and take other medicines that increase your risk of bleeding, including:
Tell your doctor if you take any of these medicines. Ask your doctor or pharmacist if you are not sure if your medicine is one listed above.
Call your doctor or get medical help right away if you or your child develop any of these signs or symptoms of bleeding:
If you take XARELTO® and receive spinal anesthesia or have a spinal puncture, your doctor should watch you closely for symptoms of spinal or epidural blood clots.
Tell your doctor right away if you have:
XARELTO® is not for use in people with artificial heart valves.
XARELTO® is not for use in people with antiphospholipid syndrome (APS), especially with positive triple antibody testing.
Do not take XARELTO® if you or your child:
Before taking XARELTO®, tell your doctor about all your medical conditions, including if you or your child:
Tell all of your doctors and dentists that you are taking XARELTO®. They should talk to the doctor who prescribed XARELTO® for you before you have any surgery, medical or dental procedure.
Tell your doctor about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Some of your other medicines may affect the way XARELTO® works, causing side effects. Certain medicines may increase your risk of bleeding. See “What is the most important information I should know about XARELTO®?”
If you take XARELTO® for:
For children who take XARELTO®
XARELTO® may cause serious side effects:
The most common side effect of XARELTO® in adults was bleeding.
The most common side effects of XARELTO®in children include:
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088. You may also report side effects to Janssen Pharmaceuticals, Inc., at 1-800-JANSSEN (1-800-526-7736).
Please read full Prescribing Information, including Boxed Warnings, and Medication Guide for XARELTO®.
Trademarks are those of their respective owners. Janssen and Bayer together are developing rivaroxaban.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenUSand https://twitter.com/JanssenGlobal. Janssen Research & Development, LLC, is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
Cautions Concerning Forward-Looking Statements
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding rivaroxaban. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s Annual Report on Form 10-K for the fiscal year ended January 3, 2021, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the company’s most recently filed Quarterly Report on Form 10-Q, and the company’s subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
1 Raffini L et al. Dramatic increase in venous thromboembolism in children’s hospitals in the United States from 2001 to 2007. Pediatrics. 2009;124:1001–1008.
2 University of Rochester Medical Center. Blood Clotting Disorders in Children. Retrieved October 27, 2021 from https://www.urmc.rochester.edu/encyclopedia/content.aspx?contenttypeid=160&contentid=39
3 Children’s Hospital of Philadelphia. Staged Reconstruction Heart Surgery. Retrieved October 27, 2021 from https://www.chop.edu/treatments/staged-reconstruction-heart-surgery
4 Johns Hopkins Medicine. Bloods Clots in Children. Retrieved October 27, 2021 from https://www.hopkinsallchildrens.org/ACH-News/General-News/Blood-Clots-in-Children
5 Children’s Minnesota. Anticoagulants: Treatment of Blood Clots. Retrieved October 27, 2021 from https://www.childrensmn.org/educationmaterials/childrensmn/article/17104/anticoagulants-treatment-of-blood-clots/
6 St. Jude Children’s Research Hospital. Do You Know Anticoagulants and Vitamin K. Retrieved October 27, 2021 from https://www.stjude.org/treatment/patient-resources/caregiver-resources/patient-family-education-sheets/nutrition-dietary/anticoagulants-and-vitamin-k.html
7 Male C, Lensing AWA, Palumbo JS, et al. Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial. Lancet Haematol. 2019 November 4; 7;1:e18-e27
8 McCrindle BW, Michelson AD, Van Bergen AH, et al. Thromboprophylaxis for children post–Fontan procedure: insights from the UNIVERSE study. J Am Heart Assoc. 2021 September 24;0:e021765
*Dr. Van Bergen is affiliated with Advocate Children’s Hospital, which was provided payment for their participation in the Phase 3 UNIVERSE clinical trial.
** The Mended Hearts, Inc. was provided a grant to help support its VTE disease awareness program.
SOURCE Janssen Pharmaceutical Companies of Johnson & Johnson
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