European Commission Approves Global Blood Therapeutics’ Oxbryta for Sickle Cell Disease

The European Commission (EC) has cleared Global Blood Therapeutics’ Oxbryta (voxelotor) to treat hemolytic anemia due to sickle cell disease (SCD) in patients 12 years and older. Oxbryta inhibits hemoglobin polymerization, a process where normal red blood cells turn into sickle cells, thereby addressing the premature destruction of red blood cells and related complications. 

The EU nod makes Oxbryta the first sickle hemoglobin polymerization inhibitor approved in Europe. 

How Does Oxbryta Work?

SCD is an inherited disorder where one or both parents pass a faulty copy of the gene that makes hemoglobin to offspring. The mutated form of hemoglobin polymerizes, making red blood cells rigid, sticky and crescent-shaped. This makes it easier for them to get stuck in narrow blood vessels, impeding blood flow and oxygen supply to the body. The fragile sickle cells die prematurely, which reduces red blood levels and causes anemia. 

Oxbryta works by increasing hemoglobin’s affinity for oxygen. As oxygenated hemoglobin do not polymerize easily, Oxbryta inhibits the polymerization process and the resultant sickling and destruction of red blood cells. 

The medication is taken once-daily by mouth.  

Related Article: Sanofi’ Enjaymo Wins FDA Approval for Rare Autoimmune Anemia

Increasing Hemoglobin Levels while Maintaining a Favorable Safety Profile

The EC approval followed a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) last December, after a Phase 3 trial called HOPE showed convincing results. 

In the study, Oxbryta increased hemoglobin levels significantly and meaningfully, while also improving hemolysis outcomes in patients.  

“Treatment with Oxbryta has been shown to break the recurrent cycle of red blood cell sickling leading to their destruction – significantly improving patients’ hemoglobin levels and the blood’s oxygen-carrying capacity,” said Dr. Baba Inusa, consultant and professor of pediatric hematology, Guy’s and St. Thomas’ NHS Foundation Trust, London and chair of the National Haemoglobinopathy Panel in England.

An analysis of the complete data from the HOPE Study was published in The Lancet Haematology in April 2021.

Common side effects of Oxbryta include headache, vomiting, diarrhea, nausea, rash, fever and abdominal pain. The adverse reactions have been described as limited and temporary. 

Related Article: China’s CANbridge Reports Positive Phase 1 Data of Contender to AstraZeneca’s Complement Inhibitor

Oxbryta Approved in the US

Oxbryta was given accelerated approval by the USFDA in 2019 to treat adults and patients older than 12. Last December, the FDA expanded Oxbryta’s indication to cover patients aged 4 and older. The drug also has Breakthrough and Orphan Drug Designations. 

Net sales of Oxbryta reached $52.1 million in the third quarter of 2021, an increase of 41% year over year. The number of patients taking Oxbryta has increased each quarter since launch.

Author

Joy Lin

Without knowledge, there is no imagination. Without imagination, knowledge is meaningless