News

Agios Pharmaceuticals has reached a significant milestone in the development of their new myelodysplastic syndrome (MDS) treatment, Tebapivat, with the FDA’s recent designation of the drug as an ‘orphan drug’. …

Novo Nordisk has received Subject Expert Committee (SEC) approval from India’s Central Drug Standard Control Organization (CDSCO) to conduct Phase III clinical trials of etavopivat in the treatment of sickle …

Pharmaceutical giant Merck has announced the start of the Phase III Shorespan-007 trial to evaluate bomedemstat (MK-3543) in approximately 300 essential thrombocythemia (ET) patients globally. The study is set to …

New data from the ongoing COMMANDS trial shows Bristol Myers Squibb‘s new myelodysplastic syndrome (MDS) drug, Luspatercept (Reblozyl) may significantly improve anemia in low-risk patients with MDS. The Director of …

Agios Pharmaceuticals continues to make remarkable strides in developing new treatments for rare blood disorders. The company’s latest quarterly report for Q2 2024 showcases impressive progress for several diseases: ▪️ …

New research suggests Alexion Pharmaceuticals, Inc.‘ immunosuppressive medication, eculizumab, may safely manage hyperhemolysis syndrome (HHS) in pediatric sickle cell disease patients. A Pediatric Blood & Cancer study reported that a …

Emmaus Life Sciences reports compliance with FDA postmarketing safety regulations for Endari, its sickle cell disease treatment. After a 2.5-day inspection under the FDA’s Postmarketing Adverse Drug Experience (PADE) program, …

Novartis has reported encouraging results for its experimental SCD treatment, a “glue degrader” small molecule. In animal studies, the drug effectively increased fetal hemoglobin (HbF) levels, crucial for managing SCD …

Novo Nordisk’s experimental hemophilia A treatment, Mim8, has demonstrated positive outcomes in its Phase 3 FRONTIER-2 trial, effectively controlling bleeding. Among patients with prior preventive treatments, 66% on once-weekly and …