Topic: sickle cell anemia

oxbryta

38. Oxbryta

Global Blood Therapeutics aims to bring a “paradigm shift” to the treatment of sickle cell disease with the launch of Oxbryta.
Adakveo

35. Adakveo

Adakveo is the first targeted therapy for vaso-occlusive crises—the painful clumps of sickled red blood cells that can block the flow of oxygen to organs—in patients with sickle cell disease.
Pfizer

11. Rivipansel

2019 might be described as something of a "red-letter year" for patients with sickle cell disease overall, with two new drug approvals and positive data from gene therapy trials, but GlycoMimetics and Pfizer missed out on the celebrations.
Fail sign

2019’s top 15 clinical trial flops (and a flip-flop)

This year’s top 15 list has been drawn up to include trials whose failure we judge to have had material consequences on their sponsors and the patients for whom the drugs are being developed. The catalog includes multiple trials for aggressive brain cancer, Alzheimer’s disease, nonalcoholic steatohepatitis and heart disease.