Investigational Rett Syndrome Therapy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company AMO Pharma Limited recently announced that the U.S. Food and Drug Administration (FDA) had decided to grant Orphan Drug designation for AMO-04, and experimental product that is in development for the treatment of Rett syndrome. This disorder has limited treatment options, and the designation serves to highlight the urgency for new treatment approaches.

Rett syndrome is a genetically linked brain disorder that occurs in girls, with the first symptoms appearing anywhere from six to 18 months of age. The disorder only appears in girls because boys who have the same mutation typically die soon after they are born. Symptoms of Rett syndrome include repetitive movements, problems with speech and coordination, difficulty walking, slow growth, small head size, scoliosis, seizures, and sleeping problems. The severity of these symptoms can vary significantly. It is rarely inherited, but mostly appears as a spontaneous mutation. Previously, Rett syndrome was thought to be related to autism. Treatment is focused on alleviating symptoms, with no known cure. Most Rett syndrome patients are able to survive into their 40s with adequate care. To learn more about Rett syndrome, click here.

AMO Phara prioritizes the development of treatments for rare and debilitating diseases that have limited or no approved options for treatment. AMO-04 is classified as a glutamate modulator, and it has shown some potential in treating Rett syndrome in a mouse model of the disease.

Orphan Drug designation is reserved for therapies that are targeted at diseases that are considered rare; under the FDA definition, this is any illness or condition that affects 200,000 people or less in the U.S. A drug can only qualify if it addresses a disease with no approved treatments or could potentially offer an improvement in outcomes for patients when compared to currently available options. This designation provides several incentives, such as tax breaks, the waiving of certain fees, and a period of seven years of market exclusivity. This helps guarantee a return on investment for the company that developed the drug.

Hopefully, further trials and testing will reveal the safety and effectiveness of AMO-04 in treating Rett syndrome.


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