Sobi Announces FDA Acceptance of New Drug Application for Avatrombopag (DOPTELET®) for the Treatment of Pediatric Immune Thrombocytopenia
Avatrombopag PDUFA date set for July 24, 2025
WALTHAM, Mass., Dec. 12, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of (Sobi®), today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for avatrombopag (DOPTELET®) 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.
The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of July 24, 2025.
The FDA also accepted Sobi’s New Drug Application (NDA) for avatrombopag for oral suspension. Avatrombopag for oral suspension is a capsule where its granules are sprinkled onto a soft food or liquid to prepare for administration.
“These application acceptances for avatrombopag brings us one step closer to much-needed, additional treatment options for adolescents and children with persistent and chronic ITP,” said Jamie Freedman, MD, PhD, Chief Medical Officer, Sobi North America. “Now, there’s an opportunity to address an unmet need in our pediatric population, with the added bonus of an additional formulation, an oral suspension option that may be more suitable for some patients.”
The application is based on results from the AVA-PED-301 study, evaluating the efficacy and safety of avatrombopag for the treatment of pediatric patients with ITP of at least 6 months’ duration. The study met the primary endpoint of durable platelet response defined as the proportion of patients achieving at least six out of eight weekly platelet counts ≥50×109/L during the last eight weeks of the 12-week treatment period in the core phase in the absence of rescue medication. The alternative primary efficacy endpoint of two consecutive PC ≥50× 109 /L was also met.
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About immune thrombocytopenia
Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low numbers of platelets that results in an increased risk of bleeding. Symptoms can be highly variable and include bruising, a rash of small dots that represent small hemorrhages, bleeding from the gums or nose, blood in the urine/stools and debilitating fatigue. It is estimated that up to 100 people per million live with ITP. The disorder is considered chronic when symptoms last more than 12 months.i Long-term, primary chronic ITP is associated with increased risk of infections, bleeding episodes requiring hospitalization, hematologic malignancies, and mortality. No cure is available, and patients with chronic ITP usually relapse after various treatments and still require treatment to reduce the risk of clinically significant bleeding.
About DOPTELET (avatrombopag)
DOPTELET is an orally bioavailable, small molecule thrombopoietin receptor agonist (TPO-RA) that stimulates the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in increased production of platelets. DOPTELET does not compete with TPO for binding to the TPO receptor.
INDICATION
DOPTELET® (avatrombopag) is is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
IMPORTANT SAFETY INFORMATION
Thrombotic/Thromboembolic Complications. DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic complications in patients with chronic liver disease (0.4%; (1/274) in DOPTELET-treated patients) and thromboembolic complications in patients with chronic immune thrombocytopenia (7%; (9/128) in DOPTELET-treated patients). Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists.
Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions and acquired risk factors.
DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Monitor platelet counts, and for signs and symptoms of thromboembolic events and institute treatment promptly.
Serious Adverse Reactions
Serious adverse reaction that occurred more frequently in patients treated with DOPTELET (9%; 12/128) compared to placebo (5%; 1/22) was headache, occurring in 1.6% (2/128).
Adverse Reactions
The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis.
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 or at .
About Sobi®
Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at , and .
