A cutting-edge biopharmaceutical company that targets rare and orphan diseases said it has submitted an application to the U.S. Food and Drug Administration requesting approval for a potentially revolutionary drug to treat sickle cell disease.
Officials for Emmaus Life Sciences, Inc., said the application for an orally administered pharmaceutical grade L-glutamine treatment 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.
Emmaus is requesting Priority Review of the application.
“We would like to thank the NIH, FDA and Ajinomoto Corporation for the funding of our early work,” Dr. Yutaka Niihara, chairman and CEO of Emmaus, said in a statement. “We are also thankful to our clinicians, employees, and partners for their efforts and to our investors for funding the work.”
The news was met with enthusiasm from the local medical community.
Dr. Lanetta Bronté, president of the Foundation for Sickle Cell Research in Baltimore, said she was thrilled with news of the application, which comes as the nation observes National Sickle Cell Disease Awareness Month.
“We are hopeful that the FDA will give the application priority review so that patients can very soon take advantage of this new therapy,” Bronté said. “Patients between the ages of 15 and 25 have a sevenfold risk in mortality and this risk needs to be gotten ahead of quickly.”
Sickle cell disease is an inherited blood disorder characterized by the production of an altered form of hemoglobin which polymerizes and becomes fıbrous, 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, medical experts said.
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.
“We are pleased to submit our NDA during National Sickle Cell Disease Awareness Month, and following the formation of the Sickle Cell Disease Coalition spearheaded by the American Society of Hematology,” Niihara said. “We hope our NDA submission will result in a change of the status quo of sickle cell disease treatment.”
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, Emmaus officials reported.
The clinical trial enrolled 230 adult and pediatric patients as young as 5 across 31 experienced sickle cell disease treatment centers in the United States. No major adverse events were attributable to the treatment.
Emmaus’ sickle cell disease therapy has Orphan Drug designation in the U.S. and Europe and Fast Track designation from the FDA.
Emmaus said they also plan to submit a marketing authorization application to the European Medicines Agency.
“The Foundation for Sickle Cell Disease Research urges all pharmaceutical and biotechnology companies in the sickle cell space to work with patient focused organizations to increase awareness of new therapies available to patients,” Bronté said.