Emmaus Medical

Emmaus Life Sciences is a commercial-stage biopharmaceutical firm focused on treatments for rare and orphan diseases. The company was founded in 2003 by Dr. Yutaka Niihara, MD, MPH, to commercially develop a treatment for sickle cell disease (SCD) that he began researching in 1992 at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. Dr. Niihara, the company's Chairman and CEO, was driven to find a practical therapy to alleviate the intense pain he witnessed in his SCD patients early in his career. His research, spanning over two decades, led to the development of the company's principal product.

The company's core business revolves around the marketing and sale of its lead product, Endari®, a pharmaceutical-grade L-glutamine oral powder. This prescription therapy is indicated to reduce the acute complications of sickle cell disease, such as severe pain crises, in adult and pediatric patients aged five years and older. The U.S. Food and Drug Administration (FDA) granted Endari approval in July 2017, making it the first new treatment for SCD in nearly 20 years and the first ever approved for children with the condition. The company's revenue is generated from the sale of Endari®, which is available through specialty pharmacies with a prescription. Emmaus is actively seeking marketing approval in other global regions with significant SCD populations, and has already gained authorization in several countries in the Middle East.

Endari® works by increasing the amount of the amino acid glutamine in the blood. While the exact mechanism is not fully understood, it is believed to help restore the balance of certain molecules (NAD+/NADH) in red blood cells, which may protect them from oxidative damage. This action helps red blood cells remain flexible, improving blood flow and reducing the frequency of painful crises and hospitalizations. The product is an oral powder that is mixed with a beverage or soft food and taken twice daily. Clinical studies have demonstrated its effectiveness in reducing the frequency of sickle cell crises and hospitalizations, and it can be used either as a standalone therapy or in conjunction with hydroxyurea.

Keywords: biopharmaceutical, rare diseases, orphan diseases, sickle cell disease, L-glutamine, Endari, Yutaka Niihara, FDA approved, orphan drug, hematology, commercial-stage pharma, prescription therapy, pain crisis reduction, pediatric sickle cell treatment, pharmaceutical development, specialty pharmacy, global drug marketing, clinical trials, hereditary blood disorder, rare blood disease