On June 19, "Shine the LIght on Sickle Cell" in celebration of World Sickle Cell Day!

A New Treatment for Sickle Cell Disease!

In July, 2017, the U.S. Food and Drug Administration approved the first new treatment for sickle cell in 20 years.

Since 1998, providers have had just one medication to treat sickle cell disease – hydroxyurea – a chemotherapy medicine that has been shown to be effective in reducing painful crises. Now a second treatment has passed through the clinical studies phases successfully. Endari® is a powder that is taken by mouth, and it works by introducing glutamine into red blood cells. Glutamine—a naturally occurring amino acid, or protein building block—helps the sickle cells flow more easily and reduces the chances that they will block blood vessels and organs.

“The approval of Endari® is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children,” Yutaka Niihara, MD, MPH, Chairman and Chief Executive Officer of Emmaus Life Sciences, said in a release.

Like many medications, Endari® has potential side effects. According to the FDA, common side effects include constipation, nausea, headache, abdominal pain, cough, pain abdomen, back, chest, and extremities. However, patients who took Endari had fewer hospital visits for pain than those who took the placebo, and they also spent fewer days in the hospital.

Emmaus Life Sciences hopes to make Endari® available to the public by the end of 2017 — and at an affordable price, so that it will be covered by most insurance plans, including Medicare and Medicaid.

Talk with your provider about these two treatments to determine if they are right for you.

More Hope on the Horizon

Clinical studies happen in phases, as researchers learn more about the safety and effectiveness of the new drug for humans. Below is a list of drugs currently in development. See clinicaltrials.gov for the most accurate and recent information.

number of new drugs in phase 3 studies

Lentiglobin (BB305*),
Rivipansel (GMI1070)
Voxelotor (GBT440) *beta thalassemia

number of studies, all phases, on new or improved use of hydroxyurea

number of phase 01 sickle cell studies curently recruiting

number of phase 02 sickle cell studies curently recruiting

number of phase 03 sickle cell studies curently recruiting

Information per clinicaltrials.gov, 12/2/17

Talk to your doctor.
It's easy!

Take our “Ask Your Doctor” sheet to your physician to start a conversation about clinical studies. There are 5 questions that will help you learn more about studies in your area.

Want more information about clinical studies?

ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world. Explore 259,383 research studies in all 50 states and in 201 countries.

ClinicalTrials.gov is a resource provided by the U.S. National Library of Medicine.

The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.

FDA is an agency within the Department of Health and Human Services.

The FDA's organization consists of the Office of the Commissioner and four directorates overseeing the core functions of the agency: Medical Products and Tobacco, Foods, Global Regulatory Operations and Policy, and Operations.

CDC laboratories routinely work with some of the most deadly germs in the world – identifying health threats and conducting vital public health research. CDC constantly develops and reviews extensive laboratory guidelines and procedures to protect both the public and laboratory workers.

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Our vision is a world without sickle cell disease

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