The Food and Drug Administration approved a new medicine Friday to reduce the complications associated with sickle cell disease, a rare blood disorder.
The drug, Endari, is made by privately held Emmaus Medical and is the first new treatment for sickle cell disease to secure FDA approval in almost 20 years.
Approximately 100,000 people in the U.S. have sickle cell disease, according to the National Institutes of Health.
Sickle cell disease is a rare, inherited disorder characterized by abnormally sickle-shaped red blood cells.
Other companies working on new, novel drugs targeting sickle cell include Global Blood Therapeutics and Novartis.
As it stated in
First New Sickle Cell Drug to Launch in 20 Years
As a result, every state and the District of Columbia requires that every baby is screened for sickle cell disease or sickle cell trait.
One in 13 African-American babies is born as a genetic carrier of sickle cell, called sickle cell trait, and one in every 365 African-American children is born with sickle cell disease, according to the National Heart, Lung and Blood Institute.
This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia.
The U.S. Food and Drug Administration approved a new drug on Friday that reduces the complications associated with sickle cell disease — the first drug approved for the blood disorder in more than 20 years.
As of last year, there were a dozen other drugs still in development for sickle cell disease.
As it stated in By Zachary BrennanThe US Food and Drug Administration (FDA) on Friday granted approval to Emmaus Medical’s Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years.
Endari is approved for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
Endari is the 24th new drug approved by FDA in 2017, already two more than all of 2016.
Sickle cell disease is an inherited blood disorder, which according to the National Institutes of Health, affects approximately 100,000 people in the US, most often in African-Americans, Latinos and other minority groups.
Development of the drug was in part supported by FDA’s Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.
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