Acadia Pharmaceuticals Inc.s drug "trofinetide" has been approved by the U.S. Food and Drug Administration to treat Rett syndrome. The firm announced on Friday that this makes it the first drug for the condition to receive this approval. 

Rett syndrome is a genetic brain abnormality affecting neurodevelopment, primarily in females. According to government estimates, it affects less than 50,000 people in the U.S.

In accordance with the decision of the U.S. health authorities, trofinetide, which will be marketed under the brand name Daybue, may be used in adult and pediatric patients older than two years. The drug has a warning about diarrhea and weight loss.

Its approval comes months after the FDA rejected Acadia′s request to use Nuplazid for more extended time to treat psychosis associated with Alzheimer′s disease. According to analysts, Daybues approval would benefit the companys growth in the coming future. By the end of April, Acadia claimed it would make the drug available to patients but did not disclose the pricing information.

The medication from Acadia works as a synthetic version of the insulin-like growth factor IGF-1 and aids in transmitting nerve impulses while reducing inflammation in the neurological tissue. The company submitted its request for marketing authorization based on the results from late-stage research where the drug showed improvement in the disease′s primary symptoms compared to a placebo. The Rett Syndrome Behavioral Questionnaire and the Clinical Global Impression of Improvement assessment measures were used to gauge symptom improvement.

For a $10 million upfront payment, Acadia acquired a license from Australian pharmaceutical company Neuron Pharmaceuticals to develop and market the medicine in North America in 2018.

(Source: https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-acadias-genetic-rett-syndrome-drug-2023-03-11/)