#Sweden #Stockholm #Sobi #PNH #EHA 2026

Sobi to Showcase Cutting-Edge Data at 2026 EHA Congress in Stockholm

Swedish Orphan Biovitrum AB, commonly referred to as Sobi, is gearing up to present ground-breaking clinical and real-world data at the upcoming 2026 Congress of the European Hematology Association (EHA), set to take place from June 11 to June 14 in beautiful Stockholm, Sweden. The focus of Sobi's presentations will cover a spectrum of rare hematological conditions such as paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), primary hemophagocytic lymphohistiocytosis (HLH), and myelofibrosis.

Dr. Lydia Abad-Franch, Sobi's Head of Research and Development and Chief Medical Officer, expressed anticipation for the event, stating, "As EHA comes to Stockholm this year, we are pleased to present new clinical and real-world evidence spanning multiple rare diseases. These presentations reflect the breadth of our ongoing research and continue to build understanding of treatment outcomes in areas of high unmet need."

Key Presentations and Data Highlights

During the conference, Sobi will offer insights derived from both clinical studies and real-world experiences. Among the notable data presented are:

Oral Presentation

• - Doptelet® (avatrombopag)

- A study on the impact of protocol-mandated dose holds on platelet response durability in pediatric patients with ITP treated using avatrombopag. This post-hoc analysis will take place on June 12 from 17:15 to 18:30 CEST (Session s429).

Poster Presentations

• - Aspaveli® (pegcetacoplan)

- Findings on the sustained long-term real-world effectiveness and safety of pegcetacoplan in patients with PNH from the COMPLETE Phase 4 study, showcased during Session 2 on June 13 from 18:45 to 19:45 CEST.
- Analysis of real-world hemoglobin concentrations and transfusion burden in patients with paroxysmal nocturnal hemoglobinuria receiving complement inhibitors based on a retrospective study using US claims data. This presentation is also planned for Session 2 on June 13 from 18:45 to 19:45 CEST.

• - Doptelet® (avatrombopag)

- Results from the REAL AVA 3.0 retrospective study, focusing on avatrombopag in TPO RA–naive adults in various phases of ITP, will be presented during Session 2 on June 13 from 18:45 to 19:45 CEST.

• - Vonjo® (pacritinib)

- An analysis about the consistency of response based on baseline platelet count in patients treated with pacritinib, to be shared in Session 2 on June 13 from 18:45 to 19:45 CEST.

Implications for Rare Disease Treatment

The presented data are integral as they contribute significantly to the understanding of treatment strategies for hematologic disorders where patient needs remain inadequately addressed. Each of these therapies—Aspaveli®, Doptelet®, Gamifant®, and Vonjo®—plays an important role in transforming patient outcomes.

• - Aspaveli®/Empaveli® (pegcetacoplan) targets C3 and C3b to regulate the complement cascade, critical in immune responses linked with serious diseases. It's notably the first approved treatment for C3 glomerulopathy affecting adults and adolescents over 12.
• - Doptelet® (avatrombopag) is crucial for managing thrombocytopenia in adults and certain pediatric patients, supporting platelet count before procedures and aiding individuals with ITP.
• - Gamifant® (emapalumab) stands out as the exclusive approved anti-interferon gamma monoclonal antibody, making crucial inroads in treating HLH conditions.
• - Vonjo® (pacritinib) is indicated for myelofibrosis, especially critical for patients at risk without sufficient treatment options.

As Sobi shares these findings, the hope is to foster dialogue on enhancing care standards, unveiling more effective protocols, and ultimately improving patient quality of life in the hematology sector. This commitment underscores Sobi’s role as a leading entity in rare disease treatment innovation, with a focus on transformative therapies.