FDA Reviewing Emmaus’ New Drug Application for Oral PGLG Treatment of Sickle Cell Disease

The U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Emmaus Life Sciences for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease.

If the FDA approves this new treatment, it would be the first FDA-approved medicine available for children with sickle cell disease, and the first potentially new treatment for adults and children in nearly two decades.

Emmaus is working in the discovery, development and commercialization of novel treatments for rare diseases. The company’s research on sickle cell disease, an orphan disease, was initiated by Yutaka Niihara, MD, MPH, who is now chairman and chief executive officer of Emmaus.

The company’s Phase 3 sickle cell disease trial (NCT01179217) aimed to test the effects of L-glutamine on red blood cells of patients with sickle cell anemia or beta-0 thalassemia.

Glutamine is an amino-acid, a part of a protein, and it is a part of a person’s normal diet. It is frequently used as a nutritional supplement or as medication for a series of medical problems, such as intestinal diseases.

The trial’s primary outcomes included the number of occurrences of sickle cell crises in 48 weeks and the number of occurrences of protocol-defined sickle cell crises that occur from week 0 to week 48. That data was used to evaluate the effectiveness of oral L-glutamine as a treatment for the conditions.

According to a press release, the trial, completed in December 2013, demonstrated a reduction in the frequency of sickle cell crises and hospitalizations, as well as a reduction in consecutive days at the hospital and a decreased incidence of the dangerous acute chest syndrome.

The clinical study enrolled 230 adults and children, as young as five years old, across 31 experienced sickle cell disease treatment centers in the United States. No major adverse reactions were reported in relation to the treatment.

Emmaus is now awaiting notice from the FDA regarding its request for a priority review. Emmaus’ PGLG therapy has orphan drug designation in the U.S., orphan medicinal product designation in the European Union and fast track designation in the U.S. Emmaus also is planning to pursue a marketing authorization application to the European Medicines Agency (EMA).