Company to Seek Marketing Approval for First New Sickle Cell Treatment in Nearly Two Decades

Emmaus Life Sciences aims for September NDA

After more than 20 years of research and development, Emmaus Life Sciences has announced that it expects to submit a new drug application (NDA) to the FDA in early September for the company’s new treatment for sickle cell disease (SCD). The treatment consists of orally administered pharmaceutical-grade L-glutamine.

The NDA submission will coincide with National Sickle Cell Disease Awareness Month, which will call attention to a disorder that, according to the National Institutes of Health, is estimated to affect up to 100,000 people in the United States and 25 million people worldwide.

In December 2013, data from a phase 3 trial of oral L-glutamine demonstrated a reduction in the frequency of sickle cell crises and hospitalizations, as well as a reduction in cumulative days hospitalized and a lower incidence of a life-threatening acute chest syndrome. The clinical study enrolled 230 adult and pediatric patients 5 years of age and older at SCD treatment centers in the U.S. No major adverse events were attributed to the treatment.

The new SCD therapy has received orphan drug and fast track designations from the FDA.

Normal red blood cells contain hemoglobin A. In contrast, the bone marrow of people with SCD produces red blood cells with a different form of hemoglobin called hemoglobin S (S stands for sickle). In a person with SCD, the red blood cells, rather than remaining round, smooth, and flexible, become sickle (crescent) shaped, inflexible, and sticky as they release oxygen to other tissues in the body.

Normal red blood cells live for approximately 120 days before they are replaced with new ones. Sickle-shaped red blood cells are destroyed more quickly, in approximately 16 days, and cannot always be replaced fast enough. As a result, people with SCD are often anemic. The complications of SCD occur when sickle-shaped red blood cells block veins, which can cause pain in the arms, legs, back, stomach, bones, skin, and other parts of the body. Tissue that does not receive normal blood flow eventually becomes damaged.

Currently there is no universal cure for SCD.

Sources: PR Newswire; August 22, 2016; and Emmaus Life Sciences; 2016.