The label now indicates hydroxyurea use does not affect benefit received from the medication.
The US Food and Drug Administration (FDA) has approved an updated label for Endari, which now indicates that the sickle cell medication still provides clinical benefit to patients regardless of hydroxyurea usage. Furthermore, the label includes a new step-by-step instruction for use section following the end of the prescribing information.
"This label update provides important information to help clinicians make informed decisions on the use of Endari," said Yutaka Niihara, M.D., M.P.H., Chairman and Chief Executive Officer of Emmaus in a statement.
"We are particularly pleased with the FDA's acknowledgement that the clinical benefit of Endari is not affected by hydroxyurea use. That acknowledgement reinforces and supports the use of Endari as a monotherapy or in combination with hydroxyurea as important treatment options for patients with sickle cell disease."
In July 2017, Endari was approved by the FDA as a medication to reduce acute complications in patients with sickle cell disease. In 2018, the drug became available by prescription in the United States.
Endari, a prescription grade L-glutamine oral powder, is indicated for pediatric patients ≥5 years of age. The most common side effects observed in clinical trials were constipation, nausea, headache, abdominal pain, cough, pain in extremities, and chest pain.
Adverse reactions leading to treatment discontinuation included 1 such case of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash.
Sickle cell disease remains a significant unmet need, affecting roughly 1 hundred thousand patients in the US and a million across the globe. Recent efforts have been made to address disparities in care among such patients, the majority of which are of African descent.