{"id":54738,"date":"2023-03-11T04:03:18","date_gmt":"2023-03-11T03:03:18","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older\/"},"modified":"2023-03-11T04:03:18","modified_gmt":"2023-03-11T03:03:18","slug":"acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older\/","title":{"rendered":"Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE\u2122 (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older"},"content":{"rendered":"
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\n— First and only approved therapy for Rett syndrome, a rare, neurodevelopmental disorder, <\/i>which affects 6,000 to 9,000 patients in the U.S.1<\/sup><\/i>\n<\/p>\n

\n— Company expects DAYBUE to be available by the end of April, 2023<\/i>\n<\/p>\n

\n— Rare Pediatric Disease Priority Review Voucher granted in connection with approval<\/i>\n<\/p>\n

\n— Conference call and webcast to be held March 13, 2023 at 8:30 a.m. Eastern Time<\/i>\n<\/p>\n

SAN DIEGO–(BUSINESS WIRE)–Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE\u2122<\/sup> (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. DAYBUE is the first and only drug approved for the treatment of Rett syndrome.\n<\/p>\n

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\n“Today marks an important milestone for the Rett community and Acadia. As the first FDA-approved drug for the treatment of Rett syndrome, DAYBUE now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome,\u201d said Steve Davis, Acadia\u2019s Chief Executive Officer. “We are grateful to all of the Rett syndrome patients, caregivers, clinical investigators and our employees who have contributed to making today a reality and look forward to getting DAYBUE to patients as quickly as possible.\u201d\n<\/p>\n

\n\u201cRett syndrome is a profoundly debilitating and complex, rare, neurodevelopmental disorder that presents differently across patients and can lead to an array of unpredictable symptoms throughout the course of a patient\u2019s life,\u201d said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center, Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center and Phase 3 LAVENDER\u2122 study investigator. \u201cNow, for the first time after decades of clinical research, healthcare providers finally have a treatment option to address a range of core behavioral, communication and physical symptoms for their patients living with Rett syndrome.\u201d\n<\/p>\n

\nRett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene.2,3<\/sup> It is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use.4<\/sup> Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.5<\/sup> Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S., <\/sup>with a diagnosed population of approximately 4,500 U.S. patients.1,6<\/sup>\n<\/p>\n

\nThe FDA approval of DAYBUE was supported by results from the pivotal Phase 3 LAVENDER study evaluating the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome five to 20 years of age. In the study, treatment with DAYBUE demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints, as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.003) at week 12. The RSBQ is a caregiver assessment that evaluates a range of symptoms of Rett syndrome including vocalizations, facial expressions, eye gaze, hand movements (or stereotypies), repetitive behaviors, breathing, night-time behaviors and mood. The CGI-I is a global physician assessment of whether a patient has improved or worsened. In the study, the most common side effects were diarrhea (82%) and vomiting (29%).\n<\/p>\n

\n\u201cThis is a historic day for the Rett syndrome community and a meaningful moment for the patients and caregivers who have eagerly awaited the arrival of an approved treatment for this condition,\u201d said Melissa Kennedy, Chief Executive Officer of the International Rett Syndrome Foundation. \u201cRett syndrome is a complicated, devastating disease that affects not only the individual patient, but whole families. With today\u2019s FDA decision, those impacted by Rett have a promising new treatment option that has demonstrated benefit across a variety of Rett symptoms, including those that impact the daily lives of those living with Rett and their loved ones.\u201d\n<\/p>\n

\nDAYBUE is expected to be available in the U.S. by the end of April, 2023.\n<\/p>\n

\nIn 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.\n<\/p>\n

\nWith the FDA approval of DAYBUE, Acadia has received a Rare Pediatric Disease Priority Review Voucher, which can be used to obtain priority review for a subsequent application.\n<\/p>\n

\nAcadia Connect\u00ae<\/sup> Offers Dedicated Patient and Family Support and Resources<\/b>\n<\/p>\n

\nAcadia Connect\u00ae<\/sup><\/b> is a multi-faceted support program that will offer personal assistance, financial resources and prescription support to patients and caregivers starting and continuing appropriate DAYBUE therapy. Each dedicated support team includes a nurse care coordinator, a family access manager and 24\/7 clinical pharmacist support. For more information, visit AcadiaConnect.com<\/a> or call 1-844-737-2223, Monday to Friday, 8 a.m. to 8 p.m. Eastern Time.\n<\/p>\n

\nConference Call and Webcast Information<\/i>\n<\/p>\n

\nAcadia management will discuss the FDA approval of DAYBUE for the treatment of Rett syndrome via conference call and webcast on Monday, March 13, 2023 at 8:30 a.m. Eastern Time. The conference call may be accessed by registering for the call here<\/a>. Once registered, participants will receive an email with the dial-in number and unique PIN number to use for accessing the call. The registration link will also be available on Acadia\u2019s website, www.acadia.com<\/a>, under the investors section and will be archived there until April 13, 2023.\n<\/p>\n

\nImportant Safety Information for DAYBUE\u2122 (trofinetide)<\/b>\n<\/p>\n

\nImportant Safety Information<\/b>\n<\/p>\n