First medicine targeting the root cause of Sickle Cell Disease

Global Blood Therapeutics announced the approval of a first-in-class therapy in sickle cell disease (SCD) for adults and children 12 years of age and older, by the U.S. Food and Drug Administration (FDA).

Oxbryta (voxelotor), an oral therapy taken once daily, is the first approved treatment that directly inhibits sickle hemoglobin polymerisation, the root cause of SCD.

“This is a major milestone for people living with SCD, their families and those who care for them.” said Ted W. Love, M.D., president and chief executive officer of GBT.

SCD affects millions of people throughout the world. SCD is a lifelong inherited blood disorder that impacts hemoglobin. The sickling process causes haemolytic anaemia and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.⁵

The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in hemoglobin levels, accompanied by reductions in red blood cell destruction (haemolysis). Data from the Phase 3 HOPE Study was published recently.

Novartis’ Adakveo (crizanlizumab) was approved recently to reduce frequency of vaso-occlusive crises in sickle cell disease.

Bluebird bio is developing LentiGlobin, a gene therapy for sickle cell disease which is in the early stages of clinical development.

Register FREE to receive the latest news, innovations and insights from Health Industry Hub; the only one-stop-hub connecting Australia’s Pharma, MedTech and Biotech industry professionals and its key stakeholders.

You may also like MSD’s PBS win triples number of lung cancer patients eligible for immuno-oncology therapy