FDA Advisory Committee to Review NDA for Endari as Treatment for Sickle Cell Disease

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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RMAT designation, CTX001

The Oncologic Drug Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Emmaus Life Sciences’s New Drug Application (NDA) for Endari to treat sickle cell disease on May 24.

“The Advisory Committee meeting is an important step forward in the review process for this promising therapy,” Yutaka Niihara, MD, chairman and CEO of Emmaus Life Sciences, said in a press release. “We look forward to engaging in a productive discussion with the Advisory Committee members and continuing to work closely with the FDA throughout the regulatory process.”

Endari is an oral pharmaceutical grade L-glutamine treatment for sickle cell anemia and sickle beta thalassemia. Glutamine is an amino acid that’s frequently used as a nutritional supplement or as medication for numerous medical problems, such as intestinal diseases.

A Phase 3 clinical trial (NCT01179217) completed in March 2014, evaluated the effects of Endari on red blood cells of 230 adults and children with sickle cell anemia or sickle beta thalassemia. The trial’s primary objectives included the number of occurrences of sickle cell crises in 48 weeks and the number of occurrences of protocol-defined sickle cell crises from week 0 to week 48.

Results showed that the treatment reduced the frequency of sickle cell crises and hospitalizations, and patients’ consecutive days at the hospital. It also decreased the incidence of dangerous acute chest syndrome. The treatment was well tolerated without causing any major adverse reactions.

If the FDA approves the NDA for Endari, it would be the first medicine approved in the United States to treat children with sickle cell disease, and the first potentially new sickle cell disease drug for adults and children in almost 20 years. Endari was granted orphan drug status in the U.S. and Europe for Endari, and received fast track designation from the FDA.

The FDA accepted the NDA for Endari in November 2016 with a target action date under the Prescription Drug User Fee Act set for July 7, 2017.

Sickle cell disease is a group of inherited blood disorders that results in an abnormality in the oxygen-carrying protein hemoglobin found in red blood cells. This leads to a rigid, sickle-like shape under certain circumstances. The complications of sickle cell disease occur when sickle-shaped red blood cells block veins, which can cause pain in the arms, legs, back and stomach, bones, skin and other parts of the body. Tissue that does not receive normal blood flow eventually becomes damaged.