Acadia seeks first-ever FDA approval in Rett Syndrome
pharmafile | April 8, 2022 | News story | Medical Communications |
Acadia has presented an update on its Phase III LAVENDER study of trofinetide for Rett Syndrome at the American Academy of Neurology’s annual meeting in Seattle. The trial, which involved around 6,000 female participants, showed statistically significant result highlighting the drug’s effectiveness and safety.
Rett syndrome is a rare and debilitating neurological disease which targets mostly females following normal development in the first six months of life. It is caused by mutations in the MECP2 gene, and occurs in one of every 10,000 to 15,000 female births worldwide. There are 6,000 to 9,000 patients diagnosed with the disease in the US.
As it stands, the FDA has not approved any treatment for Rett syndrome, so trofinetide shows promise.
Trofinetide is a synthetic analog of the amino-terminal tripeptide of IGF-1 and can reduce neuro-inflammation and support synaptic function. Having IGF-1 in the brain is critical to normal development and proper disease and injury response.
The FDA has already given trofinetide Fast Track Status and Orphan Drug Designation for Rett Syndrome. It has a Rare Paediatric Disease designation, and Acadia intends to submit a New Drug Application by the middle of 2022.
“In a severe disorder like Rett syndrome that has no approved therapies and where all of our interventions are purely symptomatic, it’s very exciting to be at a point where a Phase III trial shows benefit,” study investigator Jeffrey L Neul, MD, PhD, director of the Vanderbilt Kennedy Center and professor of paediatrics, Vanderbilt University Medical Center told Medscape Medical News.