By Hilary Rosselot

Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, announced they held a meeting with the U.S. Food and Drug Administration (FDA) to optimize the design of their Phase 3 trial. This trial is designed to support a New Drug Application (NDA) to address Fragile X Syndrome.

During the FDA meeting, Allos received invaluable and constructive feedback that is being leveraged with input from the NFXF’s Clinical Trial Committee and will be incorporated into the design of the Phase 3 trial. Allos co-founder Randy Carpenter thanked the FDA for their engagement and guidance and shared that Allos “remain[s] committed to providing a new treatment option for patients and families … and believe that arbaclofen has the potential to make a significant, positive impact on their lives.”

Hilary Rosselot, Executive Director of the NFXF shared “the Allos team has made significant progress in developing arbaclofen. The expert clinician members of our Clinical Trial Committee have provided input and guidance to optimize the design of the clinical trial and enthusiastically support performing this study. NFXF looks forward to working in partnership with Allos to better the lives of individuals with Fragile X Syndrome and their families.”

While this does not mean arbaclofen is available for you to use now, this is a big step in the right direction to continue exploring arbaclofen in FXS. Thank you to the amazing families and dedicated professionals who made this possible!

Read the full press release here.

Learn more about Allos by visiting their website:

Author Hilary Rosselot

Hilary Rosselot
Hilary joined the NFXF team in 2019. Prior to joining the NFXF team, she worked at the Cincinnati Fragile X Research and Treatment Center for over five years. She has experience as a clinical research coordinator across many types of clinical trials and served as the clinical research manager for the Cincinnati program. She earned a bachelor’s degree in psychology, a master’s, and is a SOCRA certified clinical research professional (CCRP). She enjoys time with family and friends, a great book, a strong cup of coffee and, of course, a good laugh!

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Exploring Parents’ Concerns Regarding Long-Term Support and Living Arrangements for Their Children with Fragile X Syndrome

Given the limited data regarding future planning specific to individuals with Fragile X Syndrome (FXS) and the growing population of this community, this study sought to explore the concerns and challenges caregivers of individuals affected by FXS encounter when considering long-term support plans.