FDA Grants Approval for Acadia's Powder Formulation of DAYBUE to Treat Rett Syndrome
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 12 2025
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Source: NASDAQ.COM
FDA Approval: Acadia Pharmaceuticals has received FDA approval for DAYBUE STIX, a powder formulation of DAYBUE oral solution, to treat Rett syndrome in patients aged two years and older.
Rett Syndrome Prevalence: Rett syndrome is a rare neurodevelopmental disorder that affects approximately one in every 10,000 to 15,000 female births globally.
Efficacy and Safety: The new powder formulation is expected to have the same efficacy and safety profile as the previously approved DAYBUE oral solution.
Availability Timeline: DAYBUE STIX will be available on a limited basis starting in the first quarter of 2026, with broader availability anticipated in the second quarter, while the oral solution will continue to be available.
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Analyst Views on ACAD
Wall Street analysts forecast ACAD stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for ACAD is 31.08 USD with a low forecast of 21.00 USD and a high forecast of 40.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
13 Analyst Rating
8 Buy
5 Hold
0 Sell
Moderate Buy
Current: 25.700
Low
21.00
Averages
31.08
High
40.00
Current: 25.700
Low
21.00
Averages
31.08
High
40.00
About ACAD
Acadia Pharmaceuticals Inc. is a biopharmaceutical company. It develops and commercializes medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases. It has a portfolio of product candidates and research programs that are designed to address significant unmet medical needs in CNS disorders and rare diseases. Its pipeline includes NUPLAZID (pimavanserin), DAYBUE (trofinetide), ACP-101, ACP-204, and ACP-711. NUPLAZID is a selective serotonin inverse agonist/antagonist preferentially targeting the 5-HT2A receptor. NUPLAZID is developed for the treatment for Parkinson's Disease Psychosis. Trofinetide is a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. Its early-stage research programs and product candidates include ACP-211, ACP-2591, ACP-271, and Antisense Oligonucleotide (ASO) Programs.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Acadia Pharmaceuticals to Present at 2026 J.P. Morgan Healthcare Conference
- Conference Participation: Acadia Pharmaceuticals will be represented by CEO Catherine Owen Adams at the J.P. Morgan Healthcare Conference on January 13, 2026, showcasing its innovations in neurological diseases, which is expected to attract investor interest.
- Live Webcast: The presentation will be accessible via a live webcast on Acadia's website, allowing global investors to stay updated on the company's latest developments, with an archived recording available for one month post-event to enhance transparency and investor relations.
- Product Portfolio: Acadia is committed to providing FDA-approved treatments for Parkinson's disease psychosis and Rett syndrome, demonstrating its leadership in the neurological disease sector, which may draw increased attention and investment in the future.
- R&D Pipeline: The company is developing mid- to late-stage programs targeting Alzheimer's disease psychosis and Lewy body dementia, indicating its strategic commitment to addressing underserved patient needs, potentially driving future market growth.
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Acadia Receives FDA Approval for DAYBUE STIX Treatment of Rett Syndrome
- New Drug Approval: Acadia Pharmaceuticals announced that the FDA has approved DAYBUE STIX (trofinetide) for the treatment of Rett syndrome, making it the only FDA-approved treatment, expected to provide new options for 6,000 to 9,000 patients in the U.S.
- Flexible Formulation: DAYBUE STIX features a dye- and preservative-free powder formulation that allows patients to customize dosage and taste, enhancing the medication experience for patients and caregivers and addressing the urgent need for flexibility among families.
- Clinical Validation: The efficacy and safety of the new formulation are based on results from the LAVENDER™ study, demonstrating bioequivalence to the original oral solution, ensuring that patients can expect the same therapeutic effects when using the new formulation, further solidifying Acadia's leadership in the neurological disease space.
- Market Outlook: DAYBUE STIX is expected to launch on a limited basis in Q1 2026 and more broadly in Q2 2026, marking Acadia's ongoing commitment to meet the needs of Rett syndrome patients while laying the groundwork for future revenue growth.
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