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On July 7, 2017, the U.S. Food and Drug Administration approved L-glutamine oral powder (Endari, Emmaus Medical, Inc.) for oral administration to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years and older.
Approval was based on data from a randomized, double-blind, placebo-controlled, multi-center clinical trial (NCT01179217) enrolling 230 patients (5 to 58 years old) with sickle cell anemia or sickle β0-thalassemia who had two or more painful crises within the 12 months prior to enrollment. Eligible patients stabilized on hydroxyurea for at least 3 months, continued their therapy throughout the study. Patients were randomized to receive either L-glutamine or placebo for 48 weeks followed by three weeks of drug tapering.
Efficacy was demonstrated by a reduction in the number of sickle cell crises through Week 48 among patients who received L-glutamine compared to those receiving placebo. A sickle cell crisis was defined as an emergency room/medical facility visit for sickle cell disease-related pain treated with a parenteral narcotic or parenteral ketorolac. The occurrence of chest syndrome, priapism, and splenic sequestration were considered sickle cell crises. Over the 48-week period, patients receiving L-glutamine had a median of 3 sickle cell crises compared with a median of 4 crises for those receiving placebo. Treatment with L-glutamine also resulted in fewer hospitalizations due to sickle cell pain, fewer cumulative hospital days, and a lower incidence of acute chest syndrome.
The most common adverse reactions occurring in greater than 10% of patients treated with L-glutamine were constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain. Treatment discontinuation due to adverse reactions was reported in 2.7% (n=5) of patients receiving L-glutamine. These adverse reactions included one case each of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash.
The recommended dose of L-glutamine is 10 grams to 30 grams per day (based on body weight) taken orally, twice daily. See recommended dosing based on weight listed in the product label. Each dose should be mixed in 8 oz. (240 mL) of cold or room temperature beverage or 4 to 6 oz. of food before ingestion.
Full prescribing information is available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208587s000lbl.pdf.
FDA previously granted Orphan Drug Designation to L-glutamine for this indication. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics, available at: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).
Follow the Oncology Center of Excellence on Twitter @FDAOncology.--] "FDA approved L-glutamine powder for the treatment of sickle cell disease (1)")
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As an expert in pharmaceuticals and regulatory affairs, my extensive knowledge in the field allows me to delve into the details of the article you provided. The approval of L-glutamine oral powder (Endari, Emmaus Medical, Inc.) by the U.S. Food and Drug Administration (FDA) on July 7, 2017, is a significant development in the management of sickle cell disease, particularly in adult and pediatric patients aged 5 years and older.
The approval was grounded in robust evidence from a randomized, double-blind, placebo-controlled, multi-center clinical trial (NCT01179217) that enrolled 230 patients between 5 to 58 years old. These patients had sickle cell anemia or sickle β0-thalassemia and had experienced two or more painful crises within the 12 months prior to enrollment. The trial included patients who were stabilized on hydroxyurea for at least 3 months, and they continued their therapy throughout the study.
The efficacy of L-glutamine was demonstrated by a notable reduction in the number of sickle cell crises through Week 48 among patients who received L-glutamine compared to those receiving a placebo. The definition of a sickle cell crisis included emergency room/medical facility visits for sickle cell disease-related pain treated with a parenteral narcotic or parenteral ketorolac. Over the 48-week period, patients receiving L-glutamine experienced a median of 3 sickle cell crises, while those on placebo had a median of 4 crises.
Moreover, treatment with L-glutamine resulted in additional positive outcomes, such as fewer hospitalizations due to sickle cell pain, fewer cumulative hospital days, and a lower incidence of acute chest syndrome. The most common adverse reactions associated with L-glutamine included constipation, nausea, headache, abdominal pain, cough, pain in extremity, back pain, and chest pain. Treatment discontinuation due to adverse reactions was reported in 2.7% of patients receiving L-glutamine, with specific adverse reactions detailed in the provided information.
The recommended dose of L-glutamine is 10 grams to 30 grams per day, based on body weight, taken orally twice daily. The FDA had previously granted Orphan Drug Designation to L-glutamine for this indication, highlighting its recognition as a treatment for a rare disease. Healthcare professionals are encouraged to report any serious adverse events suspected to be associated with the use of L-glutamine to the FDA's MedWatch Reporting System.
For further information, healthcare professionals and individuals interested in the details of L-glutamine's approval can refer to the full prescribing information available at the provided link: .
To stay updated on recent developments and approvals in oncology, the FDA recommends following the Oncology Center of Excellence on Twitter (@FDAOncology) and checking out their podcast, Drug Information Soundcast in Clinical Oncology (D.I.S.C.O.), available at . This podcast provides valuable insights into the latest information in the field of clinical oncology.
