US FDA approves Acadia’s Daybue for Rett syndrome treatment

SAN DIEGO, March 12: Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) has announced that the US Food and Drug Administration (FDA) has approved Daybue (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

“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,” said Steve Davis, Acadia’s 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.”

“Rett 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’s life,” 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 study investigator. “Now, 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.”

Rett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene.2,3 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 Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. Rett syndrome is believed to affect 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 US patients.

Efficacy

The 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 ImpressionImprovement (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%).

“This 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,” said Melissa Kennedy, Chief Executive Officer of the International Rett Syndrome Foundation. “Rett syndrome is a complicated, devastating disease that affects not only the individual patient, but whole families. With today’s 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.”

Available

Daybue is expected to be available in the US by the end of April, 2023.

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.

With 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.

Acadia Connect® 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.

Acadia 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. 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’s website, www.acadia.com, under the investors section and will be archived there until April 13, 2023.

Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with Daybue experienced diarrhea. In those treated with Daybue, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was of mild or moderate severity in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with Daybue.

Patients should stop taking laxatives before starting Daybue. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue Daybue if severe diarrhea occurs or if dehydration is suspected.

KUWAIT CITY, March 12: The City Clinic group proudly announces free Mega Camp for all women residing in Kuwait as part of City Clinic Group’s women’s day celebrations.

Women are the backbone of every family who balances work and home, giving very little time to tend to her health needs.

This is a golden opportunity for all such working women and home makers to take a little time off to care for their health.

Studies have shown that over 50% of women visit a doctor only if they are very ill or can’t manage with home remedies. Preventive health is last on their list as their primary commitment is to family and their wellbeing.

The City Clinic group, on the event of the Mega Gynaecology Camp presents to all women with free consultation, Laboratory investigations and scans for a day.

Date: 17.03.2023, Friday

Venue : City Clinic, Khaitan Address : Mohamed Abdullah Al Zamami Building, Block-7, IBN AL Zohar Street, 2nd Floor, Khaitan, Kuwait

Time : 9 am to 5pm

Our gynaecology specialists:

Dr. Saritha (consults in City Clinic International, Fahaheel with 25over years of experience in Kuwait)

Dr. Ambiliy (consults in City Clinic, Khaitan with over 26 years of experience in Kuwait)

Dr. Susan Sheeba Pothen (consults in City Clinic, Mahboula with over 18 years of experience in Kuwait)

Dr. Surya Devi Chelliah (consults in City Clinic Mirqab with over 16 years of experience.)

We have limited slots. Please call and pre-register on this number

WhatsApp number: 1880020

Be an early bird and have your health checked for we care for you!