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FDA grants orphan drug status to Prader-Willi treatment

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The Food and Drug Administration granted diazoxide choline, studied previously for high triglycerides, orphan drug status for the treatment of Prader-Willi syndrome. This drug is used in a diazoxide controlled release tablet known as DCCR.

“We greatly appreciate the FDA’s support of our efforts to evaluate the use of DCCR in the treatment of Prader-Willi syndrome. We are actively recruiting Prader-Willi syndrome patients for a recently initiated clinical study. Initial results from that study should be coming out during Q3 of this year,” Neil M. Cowen, PhD, president and chief scientific officer of Essentialis said in a press release from the company.

There is currently no FDA-approved treatment for Prader-Willi and, according to the release, DCCR is being studied for both Prader-Willi treatment and hypothalamic obesity.

For more information: Essentialis Obtains Orphan Drug Designation from FDA for DCCR in the Treatment of Prader-Willi Syndrome