Acadia Pharmaceuticals Submits New Drug Application to US FDA for Trofinetide for the Treatment of Rett Syndrome | New



Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome. in adults and pediatric patients aged two years. and older.

“This is an important step forward for members of the Rett community who are facing a devastating disease without approved therapies,” said Steve Davis, Chief Executive Officer. “We are grateful to the patients, their families and physicians who participated in clinical studies of trofinetide, including our pivotal Phase 3 study in lavender. We look forward to working with the FDA as they evaluate the NDA.

NDA Submission Supported by Pivotal Phase 3 Lavender Study Results study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women aged 5 to 20 suffering from Rett syndrome. The study demonstrated statistically significant improvement over placebo on the co-primary endpoints, change in the Rett Syndrome Behavior Questionnaire (RSBQ) from baseline to 12 weeks (p=0.0175; effect size = 0.37) and clinical global impression improvement (CGI-I) scale score (p = 0.0030; effect size = 0.47) at 12 weeks. The RSBQ is a caregiver’s assessment of the core symptoms of Rett syndrome and the CGI-I is a physician’s overall assessment of whether Rett syndrome is getting worse or better. In addition, the study also met its key secondary endpoint, the Developmental Profile of the Communication and Symbolic Behavior Scales. Change in Composite Infant-Toddler Checklist–Social (CSBS-DP-IT–Social) score from baseline to week 12 (p=0.0064; effect size=0.43), a scale of caregiver communication.

In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North America. Trofinetide has been granted Fast Track status and Orphan Drug Designation for the treatment of Rett Syndrome in the United States. An NDA with orphan drug designation is eligible for priority review. Trofinetide has also received Rare Pediatric Disease (RPD) designation from the FDA. With such a designation, Acadia expects to receive a priority review voucher if the NDA is approved.

About Lavender™

The Lavender Study was a phase 3, 12-week, double-blind, randomized, placebo-controlled study of trofinetide in 187 girls and young women aged 5 to 20 years with Rett syndrome, designed to evaluate its efficacy and its safety. Lavender’s co-primary endpoints included both a caregiver (Rett Syndrome Behavior Questionnaire [RSBQ]) and physician (Clinical Global Impression–Improvement [CGI-I]) Evaluation. The primary secondary endpoint was also a caregiver assessment designed to assess communication skills, the Infant-Toddler Communication and Symbolic Behavior Scales Development Profile Checklist™ – Social Composite Score (CSBS-DP- IT-Social).

About Rett Syndrome

Rett syndrome is a rare genetic neurodevelopmental disorder that occurs primarily in females after nearly normal development in the first two years of life. 1,2 It is caused by mutations on the X chromosome of a gene called MECP2. 3 Occurring worldwide in approximately 1 in 10,000 to 15,000 female births and in the United States affects 6,000 to 9,000 patients. 4 Children with Rett syndrome experience a period of developmental regression between 18 and 30 months, which is usually followed by a plateau period lasting years to decades. 1,2,5 Rett syndrome is diagnosed based on clinical assessment, usually around age three. 2.6

A complex, multisystem disorder, Rett syndrome results in profound impairment of central nervous system (CNS) function, including loss of communication skills, deliberate use of the hands, gait abnormalities, and stereotypical hand movements such as than wringing/shaking hands, clapping/tapping, mouthing and washing/rubbing automatisms. 2 People living with Rett syndrome may also experience a range of additional symptoms, such as gastrointestinal complications, skeletal abnormalities, neuroendocrine abnormalities, disruptive and anxious behaviors, as well as dysregulation of mood and sleep disorders. 1 Currently, there are no FDA-approved drugs for the treatment of Rett syndrome.

About Trofinetide

Trofinetide is an investigational drug. It is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. Trofinetide is thought to stimulate synaptic maturation and overcome synaptic and neuronal immaturities characteristic of the pathophysiology of Rett syndrome. In the central nervous system, IGF-1 is produced by the two main types of brain cells – neurons and glia. IGF-1 in the brain is essential both for normal development and for responding to injury and disease. Trofinetide has been granted Fast Track status and Orphan Drug Designation for Rett Syndrome and has also received Rare Pediatric Disease (RPD) designation from the FDA.

About Acadia Pharmaceuticals

Acadia makes breakthroughs in neuroscience to improve lives. For more than 25 years, we’ve worked at the forefront of healthcare to bring life-saving solutions to those who need them most. We have developed and marketed the first and only approved therapy for hallucinations and delusions associated with Parkinson’s disease psychosis. Our late-stage development efforts are focused on treating psychosis in patients with dementia, negative symptoms of schizophrenia, and Rett syndrome. Our early-stage development efforts focus on novel approaches to pain, cognition, and neuropsychiatric symptom management in central nervous system disorders. For more information, visit us at and follow us on LinkedIn and Twitter.

Forward-looking statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include, but are not limited to, statements regarding the timing of future events. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of these statements due to a variety of factors, including the risks and uncertainties inherent in the development, approval and commercialization of drugs. For a discussion of these and other factors, please see Acadia’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and Acadia’s subsequent filings with the Securities and Exchange Commission. . You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This cautionary statement is made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement, and Acadia undertakes no obligation to revise or update updates this press release to reflect current events or circumstances. after the date hereof, except as required by law.


1 Fu et al. Consensus guidelines for the management of Rett syndrome across the lifespan. BMJ Pediatrics Open. 2020;4:1-14.

2 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Anne Neurol. 2010;68(6):944-950.

3 Amir RE, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG binding protein 2. Nat Genet. 1999;23:185-188.

4 Acadia Pharmaceuticals Inc. Data on file. RTT US Prevalence. March 2022.

5 NIND. Rett Syndrome Fact Sheet. Retrieved from Accessed February 2022.

6 Tarquinius. Age of diagnosis of Rett syndrome: recognition patterns among diagnosticians and risk factors for late diagnosis. Pediatric neurology. 2015;52:585-591.

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CONTACT: Media contact:

Acadie Pharmaceuticals Inc.

Deb Kazenelson

(818) 395-3043

[email protected] contact:

Acadie Pharmaceuticals Inc.

Mark Johnson, CFA

(858) 261-2771

[email protected]



SOURCE: Acadia Pharmaceuticals Inc.

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PUBLISHED: 7/18/2022 4:07 PM / DISK: 7/18/2022 4:07 PM


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