FDA approves first new sickle cell drug in almost 20 years

SILVER SPRING, Md. — The Food and Drug Administration on Friday approved a new treatment for severe complications from sickle cell disease — the first in nearly 20 years for the blood disorder. Endari (L-glutamine oral powder) was developed and is manufactured by Emmaus Life Sciences

“The approval of Endari is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children,” Emmaus Life Sciences chairman and CEO Dr. Yutaka Niihara said. "Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases.”

Emmaus — which received the FDA’s orphan drug designation for Endari, whose development was partially supported by the FDA Orphan Products Grant Program — said that it was aiming to make Endari available by the fourth quarter of 2017.