Acadia Pharmaceuticals Faces Negative Trend Vote on Trofinetide Application by EMA
Acadia Pharmaceuticals "announced that the Company was informed by the Committee for Medicinal Products for Human Use - CHMP- of the European Medicines Agency of a negative trend vote on its Marketing Authorization Application (MAA) for trofinetide for the treatment of Rett syndrome, following its recent CHMP oral explanation. Subject to the outcome of the CHMP vote in February, Acadia intends to request a re-examination of the opinion by the CHMP upon its formal adoption." "While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver," said Catherine Owen Adams, CEO. "We now have more than 1,000 patients on active treatment globally, from newly diagnosed 2-year-olds to adults who have lived with their disease for decades. Our ongoing real-world experience study in the U.S. continues to show outcomes that closely mirror the impact observed in rigorous randomized clinical trials conducted across a broad age range. We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU. Our commitment to the Rett syndrome community in the EU remains steadfast, and we are fully dedicated to making trofinetide available to individuals and families who urgently need a new therapeutic option."
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EMA Committee Votes Against Acadia's Trofinetide Application
- Negative Vote on Drug Application: The EMA's Committee for Medicinal Products for Human Use voted against Acadia Pharmaceuticals' marketing authorization application for trofinetide for Rett syndrome, indicating significant hurdles for the drug's entry into the European market, which could adversely affect the company's market outlook.
- Re-examination Request Plans: Acadia intends to request a re-examination of the CHMP's opinion following its formal adoption, as EU regulations allow applicants to request this within 15 days of receiving the opinion, potentially impacting the drug's launch timeline and the company's financial performance.
- U.S. Approval Context: Despite setbacks in Europe, trofinetide has been approved in the U.S., Canada, and Israel, with the CEO emphasizing strong data supporting its efficacy in treating Rett syndrome, highlighting the drug's potential market value.
- Disappointing Clinical Trial Results: Acadia's Phase 3 COMPASS trial for intranasal carbetocin in Prader-Willi syndrome did not show statistically significant improvement on primary endpoints, which may affect the company's future R&D direction and investor confidence.
Acadia Pharmaceuticals Shares Drop Over 8% After EMA Negative Trend Vote
- Stock Price Decline: Acadia Pharmaceuticals Inc. (ACAD) saw its shares drop over 8% in after-hours trading on Monday, reflecting market concerns regarding the outlook for its drug application, particularly in the treatment of Rett syndrome.
- Negative Trend Vote: The company reported that the European Medicines Agency (EMA) committee indicated a negative trend regarding its trofinetide application, suggesting regulatory doubts about the drug's efficacy and safety, which could impact future market performance.
- Follow-Up Action Plan: Acadia intends to request a re-examination after the committee formally adopts its opinion in February next year, aiming to secure drug approval despite the significant challenges ahead.
- Market Reaction: Investors reacted strongly to this news, with the sharp decline in stock price potentially leading to a reassessment of Acadia's future strategic direction, especially in the highly competitive pharmaceutical industry.
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.
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.
FDA Grants Approval for Acadia's Powder Formulation of DAYBUE to Treat Rett Syndrome
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.
Evaluating ACADIA Pharmaceuticals' Valuation Following Significant Share Price Surge in 2024
Stock Performance: ACADIA Pharmaceuticals has seen significant stock growth, with a 58.8% increase over the past year and a current share price around $27, suggesting it may be undervalued compared to its projected intrinsic value of approximately $215.87 per share.
Valuation Analysis: A Discounted Cash Flow (DCF) model indicates that ACADIA is undervalued by 87.3%, while its Price to Earnings (PE) ratio of 17.8x is below industry averages, reinforcing the notion that the stock is attractively priced.
Narrative Approach: Investors can create personalized Narratives that connect their assumptions about ACADIA's future performance with financial forecasts, allowing for a tailored understanding of the company's fair value compared to its current market price.
Investment Caution: The article emphasizes that the analysis is general and not financial advice, encouraging investors to consider their own financial situations and objectives before making investment decisions.
EMA Committee Votes Against Acadia's Trofinetide Application
- Negative Vote on Drug Application: The EMA's Committee for Medicinal Products for Human Use voted against Acadia Pharmaceuticals' marketing authorization application for trofinetide for Rett syndrome, indicating significant hurdles for the drug's entry into the European market, which could adversely affect the company's market outlook.
- Re-examination Request Plans: Acadia intends to request a re-examination of the CHMP's opinion following its formal adoption, as EU regulations allow applicants to request this within 15 days of receiving the opinion, potentially impacting the drug's launch timeline and the company's financial performance.
- U.S. Approval Context: Despite setbacks in Europe, trofinetide has been approved in the U.S., Canada, and Israel, with the CEO emphasizing strong data supporting its efficacy in treating Rett syndrome, highlighting the drug's potential market value.
- Disappointing Clinical Trial Results: Acadia's Phase 3 COMPASS trial for intranasal carbetocin in Prader-Willi syndrome did not show statistically significant improvement on primary endpoints, which may affect the company's future R&D direction and investor confidence.
Acadia Pharmaceuticals Shares Drop Over 8% After EMA Negative Trend Vote
- Stock Price Decline: Acadia Pharmaceuticals Inc. (ACAD) saw its shares drop over 8% in after-hours trading on Monday, reflecting market concerns regarding the outlook for its drug application, particularly in the treatment of Rett syndrome.
- Negative Trend Vote: The company reported that the European Medicines Agency (EMA) committee indicated a negative trend regarding its trofinetide application, suggesting regulatory doubts about the drug's efficacy and safety, which could impact future market performance.
- Follow-Up Action Plan: Acadia intends to request a re-examination after the committee formally adopts its opinion in February next year, aiming to secure drug approval despite the significant challenges ahead.
- Market Reaction: Investors reacted strongly to this news, with the sharp decline in stock price potentially leading to a reassessment of Acadia's future strategic direction, especially in the highly competitive pharmaceutical industry.
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.
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.
FDA Grants Approval for Acadia's Powder Formulation of DAYBUE to Treat Rett Syndrome
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.
Evaluating ACADIA Pharmaceuticals' Valuation Following Significant Share Price Surge in 2024
Stock Performance: ACADIA Pharmaceuticals has seen significant stock growth, with a 58.8% increase over the past year and a current share price around $27, suggesting it may be undervalued compared to its projected intrinsic value of approximately $215.87 per share.
Valuation Analysis: A Discounted Cash Flow (DCF) model indicates that ACADIA is undervalued by 87.3%, while its Price to Earnings (PE) ratio of 17.8x is below industry averages, reinforcing the notion that the stock is attractively priced.
Narrative Approach: Investors can create personalized Narratives that connect their assumptions about ACADIA's future performance with financial forecasts, allowing for a tailored understanding of the company's fair value compared to its current market price.
Investment Caution: The article emphasizes that the analysis is general and not financial advice, encouraging investors to consider their own financial situations and objectives before making investment decisions.
