New Drug in Development for Sickle Cell Disease

Article

This is the first sickle cell disease treatment created in 20 years.

Emmaus Life Sciences, Inc, recently announced that it will submit a New Drug Application (NDA) to the FDA for their drug to treat sickle cell disease.

They expect to submit the NDA in September, which is National Sickle Cell Disease Awareness Month. The treatment is an orally administered pharmaceutical grade L-glutamine, according to a press release.

In a phase 3 trial, researchers enrolled 230 patients, which revealed that the drug was able to reduce sickle cell crises and hospitalizations. Researchers did not find that the drug caused any major adverse effect.

The treatment has already gained Orphan Drug designation in the United States and Europe, and Fast Track designation from the FDA, according to the press release.

“The submission of the NDA is the culmination of many people's life work, marking a milestone for all the patients, researchers, investors, partners, advocates and employees who have contributed to this achievement,” said Yutaka Niihara, MD, MPH, Chairman and CEO of Emmaus. “We look forward to the NDA submission early next month and discussing the positive safety and efficacy data from the trial with the FDA.”

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