FDA approves new treatment for sickle cell disease
FDA Press Announcements Jul 13, 2017
First approval for this rare blood disorder in nearly 20 years.
The U.S. Food and Drug Administration approved Endari (L–glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African–Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is approximately 40 to 60 years.
The safety and efficacy of Endari were studied in 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. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, 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 who received Endari also had fewer occurrences of acute chest syndrome (a life–threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).
Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.
Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases. In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.
The FDA granted the approval of Endari to Emmaus Medical Inc.
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The U.S. Food and Drug Administration approved Endari (L–glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African–Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is approximately 40 to 60 years.
The safety and efficacy of Endari were studied in 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. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, 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 who received Endari also had fewer occurrences of acute chest syndrome (a life–threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).
Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.
Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases. In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.
The FDA granted the approval of Endari to Emmaus Medical Inc.
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