This section highlights FDA-related milestones and regulatory updates for drugs developed by ACADIA Pharmaceuticals (ACAD).
Over the past two years, ACADIA Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ACP-711, NUPLAZID, and Trofinetide. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ACP-711 - FDA Regulatory Timeline and Events
ACP-711 is a drug developed by ACADIA Pharmaceuticals for the following indication: GABRA3 modulators.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ACP-711
- Announced Date:
- March 3, 2025
- Indication:
- GABRA3 modulators
Announcement
Acadia Pharmaceuticals Inc. announced the successful completion of the two originally planned cohorts in their Phase 1 multiple-ascending-dose MAD study (EUCT: 2024-514514-12-00) of ACP-711, formerly SAN711, in healthy volunteers.
AI Summary
Acadia Pharmaceuticals announced the successful completion of two initially planned cohorts in the Phase 1 multiple ascending dose (MAD) study (EUCT: 2024-514514-12-00) of ACP-711, formerly known as SAN711, in healthy volunteers. The study demonstrated that ACP-711 was safe and generally well tolerated, with all participants finishing the trial and no serious adverse events reported. Most of the observed adverse events were mild, and there were no issues related to safety laboratories, cardiovascular functions, or neurological assessments. Based on these promising results, Acadia plans to seek regulatory approval to extend the study by testing higher repeated doses and including elderly healthy volunteers. This advancement marks an important step in developing ACP-711 as a potential treatment for essential tremor, reflecting a positive outlook for its future clinical evaluation.
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NUPLAZID (Pimavanserin) - FDA Regulatory Timeline and Events
NUPLAZID (Pimavanserin) is a drug developed by ACADIA Pharmaceuticals for the following indication: Hallucinations and Delusions Associated with Dementia-Related Psychosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NUPLAZID (Pimavanserin)
- Announced Date:
- June 9, 2025
- Indication:
- Hallucinations and Delusions Associated with Dementia-Related Psychosis
Announcement
Acadia Pharmaceuticals Inc announced that the U.S. Court of Appeals for the Federal Circuit affirmed a decision issued by the U.S. District Court for the District of Delaware in December 2023 confirming validity of the NUPLAZID® (pimavanserin) '740 composition of matter patent.
AI Summary
Acadia Pharmaceuticals Inc. announced that the U.S. Court of Appeals for the Federal Circuit has upheld the U.S. District Court for the District of Delaware’s decision, affirming the validity of the NUPLAZID® (pimavanserin) '740 composition of matter patent. This ruling ensures that the patent remains protected until 2030. According to the court’s reasoning, an earlier filed and issued claim cannot be invalidated by a later, earlier-expiring reference claim when both share a common priority date.
The decision is a key victory for Acadia, reinforcing its intellectual property portfolio and ensuring solid market protection for NUPLAZID®. This outcome backs the company’s commitment to safeguarding its innovative therapies designed to address significant, unmet medical needs, thereby providing confidence in the strength and durability of its patent protections.
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Trofinetide - FDA Regulatory Timeline and Events
Trofinetide is a drug developed by ACADIA Pharmaceuticals for the following indication: Rett Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Trofinetide
- Announced Date:
- March 5, 2025
- Indication:
- Rett Syndrome
Announcement
Acadia Pharmaceuticals Inc. announced that the journal Medpublished results from the open-label study, DAFFODIL™, evaluating the safety, tolerability and exploratory efficacy of DAYBUE® (trofinetide) in girls ages two to four living with Rett syndrome (n = 15).
AI Summary
Acadia Pharmaceuticals announced that the journal Med published results from its open-label DAFFODIL™ study evaluating DAYBUE® (trofinetide) in girls ages two to four with Rett syndrome. In this study of 15 participants, researchers examined the safety, tolerability, and exploratory efficacy of the drug in a younger population. Findings indicated that DAYBUE maintained a safety profile similar to that observed in older patients studied in previous trials. The study offers essential insights into treatment plans and dosing strategies for children who typically begin experiencing symptoms very early in life, leading to challenges with communication and motor skills. Overall, the DAFFODIL study reinforces trofinetide’s potential role in addressing the complex needs of pediatric patients with Rett syndrome and supports its long-term use as a safe treatment option. These results help to guide future studies and clinical practices for managing this rare disorder.
Read Announcement- Drug:
- Trofinetide
- Announced Date:
- July 18, 2024
- Indication:
- Rett Syndrome
Announcement
Acadia Pharmaceuticals Inc announced that the journal Med published results from two open-label extension studies, LILAC-1™ and LILAC-2™, which showed that patients treated with DAYBUE™ who completed these studies experienced improvement in Rett symptoms as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ).
AI Summary
Acadia Pharmaceuticals announced that the journal Med recently published results from two open‐label extension studies, LILAC-1 and LILAC-2, evaluating DAYBUE treatment in patients with Rett syndrome. The studies showed that patients who completed long-term treatment with DAYBUE experienced significant improvements in their symptoms, as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ). In LILAC-1, a 40-week study following the initial Phase 3 LAVENDER trial, patients who continued on DAYBUE, as well as those who switched from placebo, saw a similar reduction in RSBQ scores. LILAC-2 further supported these findings over a 32-month period, with a majority of patients reporting at least a 10% improvement in their RSBQ scores. These results provide deeper insight into the long-term safety and potential benefits of DAYBUE for managing Rett syndrome.
Read Announcement- Drug:
- Trofinetide
- Announced Date:
- June 18, 2024
- Indication:
- Rett Syndrome
Announcement
Acadia Pharmaceuticals, Inc. announced that interim data from the open-label real-world LOTUS™ study will be presented at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting, being held this week in Westminster, Colorado.
AI Summary
Acadia Pharmaceuticals announced that interim data from its real-world LOTUS study will be shared at the 2024 International Rett Syndrome Foundation Annual Scientific Meeting in Westminster, Colorado this week. The open-label study gathers caregiver-reported insights on the effectiveness and tolerability of DAYBUE (trofinetide) in patients with Rett syndrome. Early results indicate that many caregivers observed improvements in areas such as nonverbal communication, alertness, and social interaction within one month of treatment. The findings also shed light on real-life strategies used by caregivers to manage gastrointestinal side effects, including diarrhea, by adjusting doses and implementing diarrhea management plans. These interim results support the benefits seen in previous clinical trials and provide useful information for both families and clinicians managing Rett syndrome.
Read Announcement- Drug:
- Trofinetide
- Announced Date:
- April 22, 2024
- Indication:
- Rett Syndrome
Announcement
Acadia Pharmaceuticals Inc. announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia's submission.
AI Summary
Acadia Pharmaceuticals recently announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide, a potential treatment for Rett syndrome—a rare, complex neurodevelopmental disorder primarily affecting girls. Health Canada has given the submission Priority Review status, which could fast-track trofinetide’s evaluation as a treatment option for this debilitating condition. If approved, trofinetide will be the first available therapy in Canada specifically targeting Rett syndrome, offering hope to patients and families facing significant challenges due to the condition’s unpredictable symptoms.
The acceptance of the NDS and the Priority Review designation highlight the promising clinical results that support trofinetide’s effectiveness and safety. This decision underscores Health Canada’s commitment to addressing serious medical needs and represents a key step forward in improving the quality of life for those affected by Rett syndrome.
Read Announcement - Drug:
- Trofinetide
- Announced Date:
- April 17, 2024
- Indication:
- Rett Syndrome
Announcement
Acadia Pharmaceuticals Inc. announced that two oral presentations featuring DAYBUE™ (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.
AI Summary
Acadia Pharmaceuticals Inc. announced two oral presentations on DAYBUE™ (trofinetide) findings for Rett syndrome at the American Academy of Neurology (AAN) 2024 annual meeting in Denver, CO. These sessions will highlight key data from recent studies that evaluate the drug’s safety, tolerability, and its potential benefits in young girls with Rett syndrome.
The first presentation will cover the final results from the DAFFODIL study, which involved girls aged two to four years. The second presentation will share insights from exit interviews with caregivers of patients from several open-label studies. Both presentations aim to shed light on the experiences with DAYBUE and provide further understanding of its effects on Rett syndrome symptoms.
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