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news.The US Food and Drug Administration (FDA) has accepted for review Emmaus Life Sciences' New Drug Application for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.
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Data from the company's Phase 3 sickle cell disease trial 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 the life-threatening acute chest syndrome.
The clinical trial enrolled 230 adult and pediatric patients as young as five years old, across 31 experienced sickle cell disease treatment centers in the U.S. No major adverse events were attributable to the treatment. If approved, this represents the first potential treatment for pediatric patients with sickle cell disease, and the first potential new treatment in nearly 20 years for adult patients.
The company is awaiting notice from the FDA regarding its request for a priority review. Emmaus' sickle cell disease therapy has Orphan Drug designation in the U.S., Orphan Medicinal Product designation in the EU and Fast Track designation from the FDA. Emmaus also plans to submit a marketing authorization application to the European Medicines Agency.
About Sickle Cell Disease
Sickle Cell Disease is an inherited blood disorder characterized by the production of an altered form of hemoglobin which polymerizes and becomes fibrous, causing red blood cells to become rigid and change form so that they appear sickle shaped instead of soft and rounded. Patients with Sickle Cell Disease suffer from debilitating episodes of sickle cell crisis, which occur when the rigid, adhesive and inflexible red blood cells occlude blood vessels.
Sickle cell crisis causes excruciating pain as a result of insufficient oxygen being delivered to tissue, referred to as tissue ischemia, and inflammation. These events may lead to organ damage, stroke, pulmonary complications, skin ulceration, infection and a variety of other adverse outcomes.
Sickle Cell Disease is an orphan disease in the U.S., affecting approximately 100,000 patients in the U.S. and millions worldwide with significant unmet medical needs.