Oral powder reduces sickle cell disease crises

By Sola Ogundipe

THERE is good news for persons living with Sickle Cell Disease, SCD, a rare, inherited blood disorder that causes crippling pain and shortened lifespans.

A new powder formulation known as Endari (L-glutamine oral powder)  for    treatment of the debilitating disorder has been approved by the Food and Drug Administration (FDA).

The new formulation is  approved for patients aged 5 years and older to reduce severe complications associated with the blood disorder.

Sickle cell disease affects millions of people across the world, especially those of African descent and Nigeria has the largest number of persons living with the disease in the world.

About 25 per cent of adults throughout the country have the sickle cell trait, AS, while an estimated 150,000 babies are born with sickle cell disease in Nigeria every year.

A person with sickle cell disease has red blood cells that are hard, sticky, and sickle-shaped. These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.

Painful crises are a common feature of sickle cell disease and Endari (L-glutamine) has been shown to reduce the number of crises in patients.

Several treatments have been mooted in the past for sickle cell disease, including an experimental gene therapy that is offering hope to those who suffering from the disorder.

Dr Richard Pazdur, the Acting Director of the Office of Hematology and Oncology Products in the FDA’s Centre for Drug

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