News

New findings from the ongoing phase 2/3 trial of osivelotor (GBT021601) by Global Blood Therapeutics/Pfizer highlight its potential to improve red blood cell health in sickle cell patients. Presented at EHA …

Vascarta Inc.’s VAS-101 (Vasceptor), an innovative topical curcumin treatment, has just received Orphan Drug Designation from the FDA. This status could fast-track its development, offering hope for safer, more effective …

New findings from the RESPONSE trial confirm Novartis‘s ruxolitinib provides comprehensive benefits for polycythemia vera (PV) patients who don’t adequately respond to hydroxyurea. The oral JAK1/2 inhibitor not only maintains …

Sanofi has received FDA orphan drug designation for rilzabrutinib, an investigational oral BTK inhibitor, as a potential treatment to reduce vaso-occlusive crises in sickle cell disease (SCD). The therapy is …

Emmaus Life Sciences continues to expand the reach of Endari® (L-glutamine oral powder), its FDA-approved therapy for reducing acute complications of sickle cell disease (SCD). Currently available in the U.S., …

AgiosPharma is gearing up for topline data from its Phase 3 RISE UP trial (NCT05031780), evaluating mitapivat for sickle cell disease (SCD), by year-end. The oral PK activator aims to …

Fulcrum Therapeutics is advancing its oral therapy, pociredir, for sickle cell disease (SCD) in the PIONEER #trial (NCT05169580). The study is testing safety, effectiveness, and how the drug works in …

Agios Pharmaceuticals is on track to deliver a major milestone in rare disease therapy, with the U.S. FDA decision for PYRUKYND® (mitapivat) in thalassemia expected by September 7, 2025. If …

Vascarta Inc. has initiated a Phase 1 clinical study for VAS-101, a transdermal curcumin gel designed to provide non-opioid pain relief for sickle cell disease (SCD). Conducted by the Foundation …