Blood Disorders

The European Commission has approved Swedish Orphan Biovitrum AB (OTC: BIOVF) - Apellis Pharmaceuticals Inc's (NASDAQ: APLS) Aspaveli (pegcetacoplan) for paroxysmal nocturnal hemoglobinuria (PNH). The approval covers anemic patients after treatment with a C5 inhibitor...

AstraZeneca Plc (NASDAQ: AZN) said that the European Medicines Agency (EMA) has recommended approving a medicine from its recently acquired Alexion for a rare, deadly blood disorder in children and adolescents aged up to 18 years. Ultomiris has been given a positive opinion for...

BioCryst Pharmaceuticals Inc (NASDAQ: BCRX) has announced the designs for REDEEM-1 and REDEEM-2, two upcoming pivotal trials for its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH). REDEEM-1 will compare the efficacy and safety of BCX9930...

Karyopharm Therapeutics Inc (NASDAQ: KPTI) announced updated data from Phase 1/2 trial of eltanexor (ATG-016) for patients with hypomethylating agent (HMA) refractory myelodysplastic syndrome (MDS). Data were presented at the 2021 American Society of Clinical Oncology Annual Meeting...

Apellis Pharmaceuticals Inc (NASDAQ: APLS) and Swedish Orphan Biovitrum AB (SOBI) have reported positive top-line results from the Phase 3 PRINCE study evaluating Empaveli (pegcetacoplan) in treatment naïve paroxysmal nocturnal hemoglobinuria (PNH) patients...

Global Blood Therapeutics Inc (NASDAQ: GBT), a clinical-stage biopharmaceutical company that develops therapeutics to treat blood-based disorders, received a breakthrough therapy designation in early January that has at least one analyst optimistic as to what will come next. The Analyst William...

Global Blood Therapeutics Inc (NASDAQ: GBT), a clinical-stage biopharmaceutical company that focuses on treating blood-based disorders, released results from multiple data presentations during the 59th American Society of Hematology Annual Meeting & Exposition. The Analyst Morgan Stanley'...