Hematology Month in Review: June 2025

Despite issuing no major approvals of hematology medications, the US Food and Drug Administration (FDA) granted several Orphan Drug and Fast Track designations to various drugs throughout June, from a hereditary hemorrhagic telangiectasia treatment to an oral drug for chronic neutropenia. Additionally, multiple drugs were successful in their respective studies, with iptacopan achieving its primary endpoint in a phase 3 trial and denecimig proving its tolerability in hemophilia A after switching from emicizumab.

FDA News

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

On June 12, 2025, the FDA granted Orphan Drug designation to VAS-101 for sickle cell disease. A patented topical curcumin formulation delivered through parent company Vascarta’s patented transdermal technology, VAS-101 is intended to fill the niche caused by limited bioavailability and effectiveness of oral curcumin dosing.

FDA Grants Fast Track Designation to Mavorixafor for Chronic Neutropenia

Mavorixafor, previously approved for the treatment of WHIM syndrome in 2024, has now received Fast Track designation to treat chronic neutropenia, granted by the FDA on June 10, 2025. The only other medication approved for CN, a human recombinant granulocyte-colony stimulating factor, causes several side effects, such as thrombocytopenia, osteoporosis, vasculitis, and increased risk of leukemia. Mavorixafor aims to fill this void, as few patients receive the optimal dose recommendation of the G-CSF medication.

FDA Grants Orphan Drug Designation to DIAG723 for Treatment of HHT

Announced on June 18, 2025, the first-in-class antibody DIAG723, which aims to restore ALK1 signaling and promote vascular quiescence in patients with hereditary hemorrhagic telangiectasia, also received Orphan Drug designation from the FDA. Parent company Diagonal Therapeutics has begun a natural history study of adults with HHT to characterize variability of patient-reported outcomes such as epistaxis, hematologic support, and quality of life.

New Hematology Research

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

Announced by parent company Beam Therapeutics at the European Hematology Association 2025 Congress, BEAM-101 has established a strong clinical profile. The one-time treatment, administered via a hematopoietic stem cell transplant, is designed to trigger increased production of non- and anti-sickling fetal hemoglobin.

Denecimig Well-Tolerated in Hemophilia A After Switching from Emicizumab

The phase 3b FRONTIER5 trial – fifth and final trial in the ongoing phase 3 FRONTIER program – demonstrated switching to denecimig from emicizumab can be done safely without a washout period or loading dose. Parent company Novo Nordisk also announced its intention to submit denecimig for US and EU regulatory review later in 2025.

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

APPULSE-PNH aimed to investigate the safety and efficacy of Novartis’s twice-daily oral monotherapy iptacopan in adults with paroxysmal nocturnal hemoglobinuria. On June 12, 2025, Novartis announced that patients treated with iptacopan achieved Hb ≥12 g/dL and clinically meaningful fatigue improvements without requiring transfusion.