Experimental Therapy Shows Promise for Excessive Sleepiness in Prader-Willi Syndrome

According to a story from prnewswire.com, the pharma company Harmony Biosciences Holdings, Inc., has recently announced the release of data from its phase 2 clinical trial. This trial is evaluating pitolisant as a treatment for an often overlooked but quite serious symptom of Prader-Willi syndrome: excessive daytime sleepiness. The data from the trial has been positive. The company is focused on developing innovative therapies for rare neurological illnesses.

About Prader-Willi Syndrome

Prader-Willi syndrome is a genetic disorder which is most characterized by childhood obesity that results from an abnormal, insatiable appetite. This obesity often continues into adulthood. In most cases, the syndrome is caused by the deletion of a certain section of chromosome 15. In about a quarter of cases, the patient receives two copies of chromosome 15 from the mother but gets none from the father. This syndrome is not considered heritable, as the genetic changes occur during gestation. Symptoms of Prader-Willi syndrome include slow development, poor feeding, muscle weakness, obesity, over-eating, abnormal flexibility, scoliosis, sleeping excessively, speech delays, intellectual disability, poor muscle tone, delayed puberty, and infertility. Excessive eating also leads to elevated risk of diabetes. Management may include physical, occupational, and speech therapy, limiting access to food, and injections of growth hormone (in child patients only). To learn more about Prader-Willi syndrome, click here.

About the Trial

65 patients were participating in the clinical trial with 91 percent completing the treatment to the end. 64 of the patients chose to continue into the open label extension portion. The patients were divided into three different groups based on age: children, adolescents, and adults.

The child and adult groups saw statistically significant improvements in patients that received the treatment versus placebo. For reasons that are unclear, there was an unusually high placebo response in the adolescent group. The patients which responded the most received the highest dose of the medication.

The high dose patients saw a 70 percent response rate, the low dose patients saw a 55.6 percent response, and the placebo rate was 52.6 percent. This is the results from the initial topline data, with the full data from the trial expected for release before the year ends. These results will include secondary outcomes and other measures. Nevertheless, these early findings are encouraging.


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