News

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 …

Emory University researchers have confirmed that hydroxyurea, a widely used oral chemotherapy drug, remains a safe and effective long-term treatment for children with sickle cell disease (SCD). A study tracking …

AB Science has received a U.S. patent for masitinib in treating sickle cell disease (SCD), with protection extending to 2040. The approval follows compelling preclinical results showing masitinib’s efficacy against …

New long-term data from the BELIEVE trial confirm that luspatercept, manufactured by Bristol Myers Squibb, provides lasting benefits for patients with transfusion-dependent beta-thalassemia, significantly reducing the need for frequent blood …