Published Date : Jul 14, 2017
In what may seem to be promising development in the treatment of a rare disease in a span of 20 years, the U.S. Food and Drug Administration (FDA) has on July 7, 2017 approved a new drug, Endari, for the treatment of sickle cell anemia. It is the only second new medicine approved by the U.S. FDA for threating complications associated with the rare disease so far and is being accorded the status of an orphan drug. The drug is manufactured by Emmaus Medical, Inc., a privately held biopharmaceutical company that has been at the forefront of conducting clinical trials for sickle cell therapeutics.
Clinical Trials Show Endari to Reduce Complications in Patients
Sickle cell anemia, popularly known as sickle cell disease, is a rare genetic blood disorder characterized by the presence of misshapen red blood cells that clog blood vessels restricting their abilities for the transport of the oxygen to the tissues.
In recent years, Emmaus has conducted extensive clinical trials and the results showed that people using the drug for 48 weeks experienced less number of hospital visits as well as reduced duration of the stay, compared to those taking placebo. In addition, the participants of the clinical study experienced fewer complications such as less incidence of acute chest syndrome, which can be life-threatening.
Advanced Gene Editing Methods to Lead to New Therapies
A number of common side effects, such as constipation, nausea, headache, abdominal pain, coughing, may hamper the demand for Endari in the coming years. Furthermore, Endari contains L-glutamine as an active ingredient, which can be easily purchased over the counter. This is likely to constrain the company’s insurance coverage for the medicine.
In another significant development that proves promising for the sickle cell disease treatments is a gene therapy being developed by bluebird bio. The prominent biotechnology company, based in Cambridge, is using advanced methods of gene editing based on clustered regularly interspaced short palindromic repeats (CRISPR) technology to treat the underlying causes of the disease.