FDA approved new treatment for Sickle cell disease (SCD) - Emmaus Life Sciences\' Endari

Publish Date : 2017-07-12

The United States Food and Drug Administration agency (FDA) has approved Emmaus Life Sciences' Endari (L-glutamine oral drug powder) to lower the serious complexities of debilitating, rare and lifelong genetic blood syndrome, referred as sickle cell disease (SCD) in pediatric and adult patients aged above five years.

Sickle cell disease (SCD) is a hereditary blood disorder. In this disease, the red blood cells are abnormally shaped which limits the blood flow in vessels and restrains O2 distribution in the body tissues that results in the organ damage and acute ache. Sickle cell disease (SCD) brings down oxygen levels in the blood and has a wide bang on the quality of life.

Around 25 million people are affected with sickle cell disease (SCD) including approximately 1 million in the United States. The majority of patients are from African-Americans, Latinos and other minority groups.

The L-glutamine oral drug powder, i.e., Endari is intended to lower oxidant harm to red blood cells by amending the latent oxidation reaction of nicotinamide adenine dinucleotide (NAD), a coenzyme which is chief regulator of oxidation.

Mr. Yutaka Niihara, chairman and chief executive officer of Emmaus Life Sciences said that Endari’s approval is a major landmark for the sickle cell patients who did not receive any improvement in treatment for almost 2 decades. Contrary to that, now for the 1st time ever since then, there is the treatment alternative for kids.

He further added that Endari has reinforced their dedication to seeking advanced and pioneer therapeutics that will aid in making the lives of patients with rare diseases better. They are grateful to Food and Drug Administration agency (FDA) for its motivating review. They are hoping to make treatment (Endari) accessible to patients by the end of this year.

Endari’s sanction is grounded on data from a 48-week long, multicentre Phase III, randomized, placebo-controlled, double-blind, clinical study measuring the results of Endari on 230 sickle cell disease (SCD) patients, including adults as well as children. The result data was achieved amongst 111 SCD patients treated with placebo and 298 SCD patients treated with L-glutamine in Phase II and III studies.

The outcomes demonstrated that the drug cut down the occurrence of sickle cell by 25 percent and need to be hospitalized by 33 percent.

Further determinations showed a decline in growing hospital days by 41 percent and bring down the frequency of ACS by 60 percent.

Patients with sickle cell disease (SCD) frequently undergo from devastating sequences of sickle cell crises, which happen when the inflexible, rigid and adhesive red blood cells block the vessels, follow-on in severe pain.