Acadia to seek first-ever FDA approval in Rett syndrome

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Published April 7, 2022

By Vanessa Doctor, RN


Shares in Acadia Pharmaceuticals rose 5.71% to $27.22 per share at the close of NASDAQ Wednesday on positive late-stage data from the study of an investigational treatment for Rett syndrome.

The company 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, demonstrated statistically significant results highlighting the drug’s effectiveness and safety.

This girl with Rett syndrome has the stereotyped hand-wringing movements.

Participants were observed in a double-blind, randomized, placebo-controlled procedure and results were measured using physician and caregiver metrics such as the Rett Syndrome Behavior Questionnaire and the Clinical Global Impression-Improvement assessment. The girls and young women were also evaluated using the Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist – Social Composite Score.

“The consistent efficacy across primary and key secondary endpoints in the Lavender study demonstrates the potential of trofinetide to treat Rett syndrome. We want to thank the patients, their caregivers, study site personnel, physicians and everyone who participated in the Lavender study for their contribution to making this milestone a reality,” said Kathie Bishop, Ph.D., Acadia’s senior vice president and chief scientific offer, in an earlier statement.

Rett syndrome is a rare and debilitating neurological disease that targets mostly females following normal development in the first six months of life. It is often misdiagnosed as cerebral palsy, autism or non-specific development delay. It is caused by mutations in the MECP2 gene and happens 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 country, which is characterized by brain function problems.

The U.S. Food and Drug Administration has not approved any treatment for Rett syndrome as of this writing, so the good news around trofinetide is incredibly encouraging. Trofinetide is a synthetic analog of the amino-terminal tripeptide of IGF-1 and can reduce neuroinflammation 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. The drug also has a Rare Pediatric Disease designation. 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 3 trial shows benefit,” study investigator Jeffrey L. Neul, M.D., Ph.D., director of the Vanderbilt Kennedy Center and professor of pediatrics, Vanderbilt University Medical Center told Medscape Medical News.

Acadia is progressing trofinetide after signing an exclusive license agreement with Neuren Pharmaceuticals to develop and commercialize the drug for Rett syndrome and other possible indications in North America in 2008. 

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