Sobi Announces U.S. Food and Drug Administration Approves Doptelet® (avatrombopag) for the Treatment of Thrombocytopenia in Pediatric Patients One Year and Older with Persistent or Chronic Immune Thrombocytopenia (ITP)
The primary endpoint was met in 27.8% of patients, confirming the efficacy in children and adolescents with persistent or chronic ITP
WALTHAM, Mass., July 25, 2025 (GLOBE NEWSWIRE) — Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the U.S. Food and Drug Administration (FDA) approved Doptelet® (avatrombopag) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a prior therapy. 1 The approval also includes a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for use in children ages one to less than six years. 1
'Doptelet represents a significant advancement in the treatment of children and adolescents with persistent or chronic ITP,' said Rachael Grace, MD, MMSc, Pediatric Hematologist and Director, Hematology Clinical Research at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and lead investigator of the AVA-PED-301 study. 'This therapy offers simple, flexible administration because it is oral, available as a tablet and now as a new pediatric sprinkle formulation and has no food restrictions. The approval of Doptelet for pediatric ITP offers families a new treatment option that can help address challenges in managing ITP in pediatric patients.'
Doptelet Sprinkle is the approved dosage form for patients aged one to less than six years, while Doptelet tablet is the approved dosage form for patients aged six and up. 1
The pediatric approval is based on results from AVA-PED-301, a global, randomized, phase 3 study evaluating the efficacy, safety, and pharmacokinetics of Doptelet in the treatment of pediatric subjects with ITP. The study showed that 27.8% of Doptelet patients achieved the primary endpoint of durable platelet response* versus 0% of placebo patients in the absence of rescue medication (p=0.0077, 95% CI 15.8-39.7). Also, 81.5% of Doptelet patients achieved the alternative primary endpoint of platelet response** versus no patients in the placebo group (p=<0.0001, 95% CI 71.1-91.8). At Day 8, 55.6% of Doptelet patients (95% CI: 41.4%, 69.1%) had a platelet count ≥50×10^9/L in the absence of rescue therapy (p<0.0001) versus no placebo patients (95% CI: 0.0%, 16.1%). Doptelet was generally well-tolerated. In pediatric patients with persistent or chronic immune thrombocytopenia, the most common adverse reactions (≥ 10%) were viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.1
'Since its introduction in 2019, Doptelet has been a cornerstone therapy for chronic ITP in adults,' said Duane Barnes, President of Sobi North America. 'This approval not only reinforces our commitment to innovation but also allows us to expand the treatment experience for patients and their families by offering Doptelet in two formulations.'
Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low numbers of platelets, leading to bruising and an increased risk of bleeding. It is estimated up to 100 people per million live with ITP. In children, ITP occurs in five out of 100,000 children per year and up to 25% of newly diagnosed pediatric ITP patients develop chronic ITP. Currently, no cure is available, and patients usually relapse after various treatments.
Indications
Doptelet is a thrombopoietin receptor agonist indicated for the treatment of: Thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.
Thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment
Thrombocytopenia in pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment
Important Safety Information
Doptelet is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or immune thrombocytopenia. In patients with chronic liver disease, thromboembolic events (portal vein thrombosis) occurred in 0.4% (1/274) of patients receiving Doptelet. In adult patients with chronic immune thrombocytopenia, thromboembolic events (arterial or venous) occurred in 7% (9/128) of patients receiving Doptelet.
Doptelet should not be administered to patients with chronic liver disease or immune thrombocytopenia in an attempt to normalize platelet counts.
The most common adverse reactions in clinical trials were viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.
To report SUSPECTED ADVERSE REACTIONS, contact Sobi, Inc. at 1-866-773-5274 or FDA at 1-800-FDA-1088 or fda.gov/medwatch.
Please see the full Prescribing Information for Doptelet.
* A durable response consisted of patients achieving a platelet count >=50,000/uL for six of the last eight weeks in the 12-week core phase of the AVA-PED-301 study.
** Efficacy was also evaluated by platelet response, defined as the proportion of subjects achieving at least two consecutive platelet assessments ≥50×10^9/L in the core phase in the absence of rescue medication.
About Immune thrombocytopenia (ITP)
Immune thrombocytopenia (ITP) is a rare, acquired autoimmune condition characterized by a low platelet count and an increased risk of bleeding, and represents a multi-faceted disease burden for children and adolescents. Bleeding can range from minor symptoms, such as bruising, to more severe findings such as intracranial hemorrhage. ITP can also have a significant impact on quality of life including severe fatigue and restrictions on physical activity due to risk of bleeding.
About Doptelet® (avatrombopag)
Doptelet is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved by the US Food & Drug Administration (FDA) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure and by the European Medicines Agency (EMA) for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. In June 2019, Doptelet was approved by the FDA for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment and in 2021, Doptelet was approved by EMA for the treatment of primary chronic ITP in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).
About Sobi North America
As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi's vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa/en or at www.sobi.com.
About Sobi®
Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here.
References
1. Doptelet Prescribing Information. Doptelet U.S. Product Information. Last updated: July 2025. Morrisville, NC: AkaRx, Inc.
Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
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