The FDA granted orphan drug designation to rilzabrutinib for warm autoimmune hemolytic anemia and IgG4-related disease.

Heme Today
Heme Today covers the latest news and analyses in bleeding, clotting, red blood cell, and iron disorders for hematologists and oncologists focused on bleeding disorders.
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In a phase 1 trial the product performed favorably in aplastic anemia, myelofibrosis, and hypoplastic myelodysplasia.
The reduced-dose oral regimen also had lower incidence of clinically relevant bleeding complications and mortality rate.
The platform features incubation technology able to replicate cell microenvironments associated with SCD sickling.
ASH Awards Commitee Chair Ivan Maillard, MD, PhD, and prior awardee Rinku Majumder, PhD, tell of the Grant's positive impact.
Bexobrutideg, an orally bioavailable, brain-penetrant BTK degrader, is being evaluated in a phase 1a/b study.
The agent recalibrates the immune system to attack tumors and increases cancer susceptibility to standard of care therapy.
Investigational T-Cell Immunotherapy Superior to Standard HSCT in Rate of Survival Plus GvHD Freedom
A phase 3 study comparing the treatments in multiple hematologic malignancies also saw greater overall survival at one year.As the most common bleeding disorder, von Willebrand disease is found in up to 1% of the United States population.
Azercabtagene zapreleucel is an allogeneic CD-19-targeting therapy combining lymphodepletion chemotherapy with interleukin-2.
The hepcidin mimetic increased hematocrit control and reduced both phlebotomy need and symptoms in a phase 2 trial.
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A phase II trial calculated a 4-year event-free survival rate of 96% and overall survival rate of 100% from the combination.
Differences were identified between patient racial groups and by urban versus rural populations.
A magnetic resonance imaging study has compared tissue iron levels and biventricular function between the two conditions.