Hematology First Half Recap: 2025

Although hematology has experienced a relatively slow year compared to other specialties, it has not been without advancements of its own. The approval of fitusiran and the Orphan Drug designations distributed by the US Food and Drug Administration (FDA) have been groundbreaking, and drugs still in the pipeline like iptacopan and ruxoprubart are moving towards regulatory decision quickly.

To mark the end of the first half of 2025, the editorial team of HCPLive Hematology created a recap of the biggest news from the last 6 months. This recap focuses on 5 critical regulatory updates from the FDA, our 5 biggest trial announcement articles, and 3 important insights from top experts featured prominently in our coverage.

First Half of 2025: Regulatory Updates in Hematology

FDA Approves Axatilimab 9 mg and 22 mg Vial Sizes for Chronic GVHD

On June 15, 2025, parent company Incyte and Syndax Pharmaceuticals announced the FDA’s approval of axatilimab-csfr in 9mg and 22mg vial sizes. The medication was initially approved in August 2024 for the treatment of graft-versus-host disease after the failure of ≥prior lines of systemic therapy in adult and pediatric patients. Axatilimab is the first and only FDA-approved therapy for chronic GVHD targeting CSF-1R to determine drivers of inflammation and fibrosis.

FDA Grants Orphan Drug Designation to DIAG723 for Treatment of HHT

On June 18, 2025, the FDA granted Orphan Drug designation to DIAG723, a first-in-class antibody treatment for hereditary hemorrhagic telangiectasia. It also received a positive opinion from the European Medicines Agency, confirming that it meets the EU’s standards for a similar designation. Parent company Diagonal Therapeutics has also begun a natural history study of adults with HHT, aiming to characterize variability of patient-related outcomes like epistaxis and quality of life.

VAS-101 (Vasceptor) Receives Orphan Drug Designation for Sickle Cell Disease

On June 12, 2025, VAS-101 also received Orphan Drug designation from the FDA for the treatment of sickle cell disease. VAS-101, a patented topical curcumin formulation delivered through parent company Vascarta’s patented transdermal technology, is intended to fill the niche made by oral curcumin’s limited bioavailability and effectiveness. The phase 1 clinical trial is ongoing, with primary endpoints of safety, tolerability, and effects on impaired blood flow dynamics.

FDA Grants Fast Track Designation to Mavorixafor for Chronic Neutropenia

On June 10, 2025, the FDA gave Fast Track designation to mavorixafor, an oral CXCR4 antagonist to treat chronic neutropenia. Previously approved in 2024 for WHIM syndrome, mavorixafor was also granted Breakthrough Therapy designation for its first submission. A phase 3 studying the agent – titled 4WARD – is ongoing.

Fitusiran Approved for People With Hemophilia A or B, With and Without Inhibitors

On March 28, 2025, fitusiran was approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A or B. Based on results from the ATLAS-OLE trial, fitusiran treatment regimens yielded a 70% reduction in mean annualized bleeding rate compared with BPA prophylaxis, while being comparable with CFC prophylaxis.

First Half of 2025: Trial Announcements in Hematology

Genetically Modified Cell Therapy BEAM-101 Successful in Sickle Cell Disease Trial

On June 23, 2025, Beam Therapeutics announced new data from the BEACON phase 1/2 clinical trial of BEAM-101, a genetically modified cell therapy for sickle cell disease. The trial is still ongoing, with both adolescent and adult cohorts anticipated to finish dosing by mid-2025 and final data expected by the end of the year.

Iptacopan (Fabhalta) Hits Primary Endpoint in Phase 3 APPULSE-PNH Trial

On June 12, 2025, Novartis announced the success of APPULSE-PNH, a phase 3b trial for the safety and efficacy of the twice-daily oral monotherapy iptacopan for paroxysmal nocturnal hemoglobinuria. The vast majority of patients achieved the goal of Hb ≥12 g/dL, and none required transfusion. Investigators also found that iptacopan maintained intravascular hemolysis control and resolved extravascular hemolysis control.

Birtamimab Misses Primary Phase 3 Endpoint for AL Amyloidosis

Birtamimab, an investigational, humanized monoclonal antibody, was designed by parent company Prothena to target and clear the amyloid leading to organ dysfunction and failure in patients with AL amyloidosis. Announced on May 23, however, birtamimab missed the primary endpoint of the phase 3 AFFIRM-AL clinical trial. Prothena has since discontinued its development.

Phase 3 Data Shows Iptacopan Eases Fatigue in C5i-Experienced, -Naïve PNH Patients

Announced on January 15, 2025, the phase 3 APPLY-PNH and APPOINT-PNH, evaluating iptacopan’s influence on fatigue and quality of life in patients with paroxysmal nocturnal hemoglobinuria, achieved their respective endpoints. Iptacopan exhibited significant improvements in both fatigue and QOL in both C5i-experienced and C5i-naïve patients.

Ruxoprubart Shows Efficacy for PNH in Interim Phase 2 Trial Results

Interim results from the phase 2 trial of ruxoprubart were announced on May 19, 2025, indicating the safety, tolerability, and efficacy of the medication in patients with paroxysmal nocturnal hemoglobinuria. These 12-week data indicated ruxoprubart’s clearing of all primary efficacy endpoints, avoiding transfusion by protecting PNH Type III cells from Alternative-Pathway-mediated destruction. Parent company NovelMed has announced plans to present these findings to regulatory authorities and begin a phase 3 trial.

First Half of 2025: Expert Perspectives in Hematology

Pediatric Year in Review: New Therapies in Sickle Cell Disease, with Shaina Willen, MD

Shaina Willen, MD, a board-certified provider in pediatric hematology/oncology and pediatric pulmonology from UC Davis Health, discusses the new therapies for sickle cell disease and the ensuing growth in treatment options for patients.

Adding Treatment Options to the Hemophilia Landscape With Fitusiran’s Approval, with Margaret Ragni, MD, MPH

Margaret Ragni, MD, MPH, professor of medicine and clinical and translational research, division of hematology/oncology, University of Pittsburgh, and director of the Hemophilia Center of Western PA, discusses the approval of fitusiran and the immense expansion of the hemophilia A and B treatment landscape that ensued.

Steven W. Pipe, MD: Supporting Gene Therapy Implementation for Hemophilia

Steven Pipe, MD, professor and Laurence A. Boxer research professor of pediatrics and professor of pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan, speaks on a discussion held at the American Society of Hematology meeting in December of 2024, in which Pipe emphasized the importance of guiding patients and colleagues through determining gene therapy eligibility.