Trofinetide Granted Rare Pediatric Disease Designation for the Treatment of Rett Syndrome

ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of Rett syndrome, a neurological disorder that sees the onset of symptoms shortly after the first six months of life. The RPD designation is given for medications that treat severe diseases that affect under 200,000 people in the United States who are under the age of 18. Researchers hope that this designation will lead to approval from the FDA.

About Rett Syndrome

Rett syndrome is a severe, rare neurological disorder that primarily affects females. It was once thought to be a form of autism, but has since been distinguished as its own disorder. It is a genetic disorder, with a mutation occurring on the X chromosome. The exact location of this mutation and its effects are unknown, but researchers do know that it is typically a sporadic mutation.

Symptoms of this condition usually appear between the first 12-18 months of life. Effects include slowed brain growth, a small head, issues with muscle coordination, social anxiety, lack of language skills, seizures, uncoordinated breathing, and a tense or irritable disposition.

After these symptoms are noticed, doctors will conduct a clinical examination and rule out other conditions, such as autism. Genetic testing will be used to confirm, and it can also show the severity of a specific case. There is no cure for Rett syndrome, and treatment consists of symptom management. Doctors will suggest physical, speech, and occupational therapy. They may also prescribe anti-seizure medications.

About Trofinetide

Trofinetide is a “novel synthetic analog of the amino‐terminal tripeptide of IGF-1”, and it is intended to reduce the inflammation in the nervous system and help the function of the synapses. If successful, it treats the major symptoms of Rett syndrome.

The FDA has granted this medication multiple designations, including Rare Pediatric Disease, Orphan Drug, and Fast Track Status. These designations have supported the third phase of Trofinetide’s clinical trial. The LAVENDER and LILAC studies will last for 12 weeks and span over at least 11 study sites. They are double-blind, placebo-controlled, long-term, and open-label.

Medical professionals are excited about these designations and are hopeful that Phase 3 will show positive results. Rett syndrome currently has an unmet need, and they hope that Trofinetide will address this need.

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