Hematology

Stay updated with the latest healthcare breakthroughs, including FDA approvals and other regulatory updates, in this week’s essential news roundup.

This updated approval builds on the success of Fibryga’s 2024 approval for AFD, which was based on positive data from the phase 3 FIBRES trial.

The updated guideline offers recommendations for RBC transfusions and iron management, while continuing to prioritize patient-centered care.

The decision expands upon caplacizumab’s 2019 approval for adults with acquired thrombotic thrombocytopenic purpura.

The NDA is based on positive 52-week data from the phase 3 VERIFY clinical trial, which demonstrated rusfertide’s capacity to reduce phlebotomy dependency.

Catch up with major FDA decisions, critical conference news, and more.

The FDA has approved small-molecule pyruvate kinase R activator mitapivat for the treatment of anemia in adults with alpha- or beta-thalassemia.

The approval indicates narsoplimab as the first and only treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy.

Stay updated with the latest healthcare breakthroughs, including FDA news and phase 3 trial readouts, in this week's essential news roundup.

Grifols’ fibrinogen concentrate is now approved for acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency.

FibroGen’s oral HIF-PH inhibitor showed improved transfusion independence in high–high-transfusion-burden MDS patients in a post hoc phase 3 MATTERHORN analysis.

Kuter discusses the efficacy and safety of rilzabrutinib in sustaining platelet responses and symptom improvement over the LUNA3 long-term extension.

Stay updated with the latest healthcare breakthroughs, including several new FDA approvals, in this week's essential news roundup.

Jain describes the phase 3 WIL-33 trial, which marked the first test of pdVWF/FVIII in adolescent patients aged <6 years.

A prediction model ties emergency visits and opioid doses with return visits in children with sickle cell disease pain.

Zackon discusses a retrospective 20-year analysis, which implies an easier-to-achieve hemoglobin target in myelodysplastic syndrome treatment.

Jang discusses the results of an open-label extension highlighting the comparative efficacy of this combination therapy versus standard-of-care ravulizumab.

End-of-study analysis from the HOPE-B trial shows durable endogenous FIX expression, reduced annualized bleeding rates, and a favorable safety profile.

The approval makes etuvetidigene autotemcel (Waskyra) the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome.

George highlights bezuclastinib’s effect on bone marrow systemic mastocytosis, emphasizing the drug’s potential in reducing symptom burden.

Data from VAYHIT3 presented at ASH 2025 suggest efficacy and safety of ianalumab in patients with primary immune thrombocytopenia.

Morris discusses this latest trial in a long list of phase 3 studies which failed to evaluate arginine therapy’s value in vaso-occlusive pain episodes in SCD.

Al-Samkari discusses the positive results from the VAYHIT2 trial, which investigated ianalumab in combination with eltrombopag in adults with ITP.

Gwarzo discussed the shortcomings faced by many patients with SCD during pain crises, due to less frequent opioid prescription than guidelines recommend.

Announced by Cogent Biosciences, these data indicate bezuclastinib’s capacity for improving patient-reported symptoms of mast cell burden.