US approves first sickle cell treatment in 20 years

MaxCyte and NIH to develop therapies for sickle cell disease
Credit: Romaset

The US Food and Drug Administration has green lit Endari, the first sickle cell treatment approved in the US for peaditaric patients, and the first for adults in 20 years.

Endari is intended to reduce the severe complications of sickle cell disease (SCD) in adult and paediatric patients age 5 and older.

The treatment reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide, a coenzyme that has been identified as the primary regulator of oxidation.

“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,” said Yutaka Niihara, MD, MPH, Chairman and CEO of Emmaus Life Sciences.

SCD is a rare, debilitating and lifelong hereditary blood disorder that affects approximately 100,000 patients in the US and up to 25 million patients worldwide, the majority of which are of African descent as well as Latinos and other minority groups.

Approximately one in every 365 African American children is born with SCD and children between the ages of 2 and 7 are 400 times more likely to suffer from stroke.

Caused by a genetic mutation in the beta-chain of hemoglobin that distorts red blood cells into crescent shapes, SCD lowers oxygen levels in the blood and has an extensive impact on morbidity, mortality and quality of life.

Patients often suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells block the blood vessels, resulting in excruciating pain.

Sickle cell crises can lead to organ damage, stroke, pulmonary complications, and other adverse outcomes, including acute chest syndrome, which may be potentially fatal and is the leading cause of death among people with SCD.

Theapproval was supported by efficacy data from a 48-week randomised, double-blind, placebo-controlled, multicenter Phase 3 clinical trial evaluating the effects of Endari, prescription grade L-glutamine, as compared to placebo on 230 adults and children with SCD.

The results demonstrated that Endari reduced the frequency of sickle cell crises by 25% and hospitalizations by 33%. Additional findings showed a decrease in cumulative hospital days by 41% and lower incidence of ACS by more than 60%.