FDA Approves 1st Drug for Sickle Cell Disease in Nearly 20 Years

July 12, 2017

FDA Approves Endari for Sickle Cell Disorder

On Friday, July 7th, the FDA approved Endari (L-glutamine oral powder) to reduce the complications associated with sickle cell disease in patients age five years and older.

What is Sickle Cell Disease?

Sickle cell disease is a rare, inherited blood disorder “in which the red blood cells are abnormally shaped (in a crescent, or "sickle," shape). This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage.”  According to the NIH, sickle cell occurs most often in African-Americans, Latinos, and other minority groups; furthermore, NIH notes that about 100,000 people in the US suffer from the condition.  Typically, people with the disease live for about 40 to 60 years.

FDA Approves Endari

Endari, manufactured by Emmaus Medical Inc., was approved by the FDA last week.  In seeking approval from the Agency, Emmaus studied the safety and efficacy of Endari by conducting “a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial.”  During this trial, Emmaus found that, when compared to patients who received a placebo, patients being treated with Endari experienced:

  • fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises)
  • fewer hospitalizations for sickle cell pain
  • fewer days in the hospital
  • fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease)

Like all drug products, Endari does not come without the possibility of side effects.  According to FDA’s recent announcement, “common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.”

The product received Orphan Drug Designation, “which provides incentives to assist and encourage the development of drugs for rare diseases.”  Additionally, the FDA Orphan Products Grants Program supported the development of Endari.  This program “provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.”

Monumental Approval

The approval of Endari is a big deal for not only the FDA and Emmaus Medical, but also patients suffering from sickle cell disease and their families.  This is because it is the first drug to be approved for this rare blood disorder in almost 20 years.  In FDA’s recent statement, Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence states that “until now, only one other drug was approved for patients living with this serious, debilitating condition."

Are you developing an orphan drug or other FDA-regulated product? Our regulatory group can help get your drug the desired FDA approval.  To learn more about our services and how we can help you, contact us today.


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