Avatrombopag Maleate

Avatrombopag PDUFA date set for July 24, 2025WALTHAM, Mass., Dec. 12, 2024 (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) 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. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here. About immune thrombocytopeniaImmune 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 https://www.sobi.com/usa or at www.sobi.com. 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 sobi.com, LinkedIn and YouTube.

STOCKHOLM, Dec. 3, 2024 /PRNewswire/ -- New data from Sobi® and partners will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA (USA) from the 7th - 10th of December 2024. During the meeting several analyses will be presented on haemophilia A, haemophagocytic lymphohistiocytosis (HLH), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and immune thrombocytopenia (ITP). "Haemophilia A has a lasting impact on joint health and quality of life. At ASH 2024, three oral presentations will reveal important new data about long-term outcomes with ALTUVOCT® prophylaxis," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We will also present new data spanning different severe and debilitating rare diseases, including an oral presentation on the real-world effectiveness of Doptelet® in treating people with ITP, demonstrating our broader commitment to advancing innovative treatments for people with rare haematological diseases. We look forward to sharing these findings in San Diego." Key data to be presented at ASH 2024 About ALTUVOCT® ALTUVOCT® (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. ALTUVOCT builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019. It is approved and marketed as ALTUVOCT by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Gamifant® Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Vonjo® Vonjo (pacritinib), a JAK-1 sparing JAK2/IRAK1/ACVR1 inhibitor, is approved for the treatment of adults with intermediate- or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L in the United States. This indication is approved under FDA accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Currently, a Phase 3 study (PACIFICA) is being conducted to assess pacritinib in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. About Aspaveli®/ Empaveli® Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the United States, European Union, and other countries globally. Currently, a Phase 3 trial (VALIANT) is being conduted to assess the efficacy and safety of pegcetacoplan in patients with C3G and primary IC-MPGN. Aspaveli/Empaveli is also under investigation for several other rare diseases across haematology and nephrology. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® Doptelet (avatrombopag) 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 in over 30 countries worldwide, including the EU and the US, for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100,000 adults per year with a prevalence of 9.5 per 100,000 adults. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, 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 sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED