FDA Accepts New Drug Application for Sickle Cell Treatment

Emmaus Life Sciences recently announced that the FDA has accepted for review the New Drug Application for its novel sickle cell disease treatment.

The drug is an oral pharmaceutical grade L-glutamine used to treat patients with the blood disorder. Patients with sickle cell disease have a variation of hemoglobin that causes blood cells to change form, and become sickle shaped rather than rounded.

These patients experience episodes of sickle cell crisis, which happens when the rigid blood cells cluster in blood vessels, according to a press release from Emmaus. Patients experience extreme pain due to a lack of oxygen being delivered to tissues.

Without treatment, sickle cell crisis can cause organ damage, stroke, lung complications, ulceration of the skin, infection, and multiple other events.

The FDA has accepted Emmaus’ application for pharmaceutical grade L-glutamine due to positive results from a phase 3 clinical trial. Included in the clinical trial were 230 patients age 5 and older.

Investigators discovered that the drug was able to reduce the amount of sickle cell crises and hospitalizations experienced by these patients, according to Emmaus. They also found that there was a reduction in days hospitalized, and patients also had a lower risk of acute chest syndrome.

There were no major adverse events reported during the clinical trial. If the FDA approves this treatment, pharmaceutical grade L-glutamine would become the first treatment available for pediatric patients, and the first new treatment in 20 years for adults.

Emmaus is currently waiting to hear back from the FDA if the drug was accepted for a priority review. The drug has already gained Orphan Drug designation in the United States and Europe, and Fast Track designation from the FDA for treating patients with sickle cell disease.

Emmaus also is planning to submit a marketing authorization application to the European Medicines Agency, the company said. Earlier this year, Emmaus joined patient advocacy groups and other stakeholders to create the Sickle Cell Disease Coalition to improve many aspects of disease care, such as access to treatments, training and education, research, clinical trials, and overall health.

The approval of this new treatment would likely accomplish many of the goals set by the Coalition.

“Around the world, people with sickle cell disease face unique challenges. In countries without early screening programs, patients are at risk for early death and childhood stroke. Due to the lack of access to appropriate care in the United States, people with sickle cell disease still face a lifetime of mental and physical disability. We can no longer accept that this is the status quo,” said Charles S. Abrams, MD, in a previous press release from Emmaus. “The Sickle Cell Disease Coalition is bringing together a number of groups that care about this disease and are committed to harness opportunities to effectuate change.”