Emmaus Life Sciences Announces Availability of Endari™ (L-glutamine Oral Powder) for Sickle Cell Disease in the United States
January 17, 2018 at 09:00 am
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Emmaus Life Sciences announced that Endari is now available by prescription in the United States. Endari was approved by the U.S. Food and Drug Administration (FDA) to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. Endari is the first new treatment approved by the FDA for pediatric patients 5 years of age and older with sickle cell disease, and the first new treatment approved by the FDA in nearly 20 years for adult patients. FDA’s approval of Endari is based on safety and effectiveness data from a randomized, double-blind, placebo-controlled, multi-center clinical trial of 230 patients ages 5 to 58 years old with sickle cell disease who had 2 or more painful crises within 12 months prior to enrollment. Patients who were treated with Endari over a 48-week period experienced fewer crisis episodes compared to patients who received a placebo (median 3 vs median 4); fewer hospitalizations for sickle cell pain (median 2 vs median 3), and fewer days in the hospital (median 6.5 days vs median 11 days). Patients on Endari also had fewer occurrences of acute chest syndrome, a life-threatening complication of sickle cell disease (8.6% vs 23.1%). A sickle cell crisis was defined as a visit to an emergency room/medical facility for sickle cell disease-related pain treated with a parenterally administered narcotic or ketorolac; the occurrence of chest syndrome, priapism, and splenic sequestration were also considered sickle cell crises.
The most common side effects of Endari seen during the study included constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain. Many of these side effects were also seen in patients taking placebo. Emmaus continues to work actively with payers to ensure coverage and access for patients with sickle cell disease.
Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company. The Company is engaged in the discovery, development, marketing, and sale of treatments and therapies, primarily for rare and orphan diseases. The Companyâs lead product, Endari (prescription-grade L-glutamine oral powder), helps to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients aged five years and older. Its product pipeline includes ELS001/ELS007, ELS004, ELS005, ELS003, and ELS002. The Company, through Kainos Medicine, Inc. (Kainos), has a license in the territory encompassing the United States, the United Kingdom, and the European Union to patent rights, know-how, and other intellectual property relating to Kainosâs IRAK4 inhibitor, referred to as KM10544, for the treatment of cancers, including leukemia, lymphoma, and solid tumor cancers. It has also developed chondrocyte and osteoblast cell sheets using human mesenchymal stem cells.