Today, Bridge the Gap - SYNGAP Education and Research Foundation announced they will host the first SYNGAP1 Patient-Focused Drug Development Meeting - SYNGAP1: Patient Voices. It will be held on November 19, 2020 in conjunction with their International SYNGAP1 Conference at the NIH/NINDS Neurological Center in Rockville, Maryland. It will be followed the next day by a SYNGAP1 Industry Round Table. This will be the first FDA Patient-Focused Drug Development Meeting in the history of the program to be held at the the National Institutes of Health.
Bridge the Gap - SYNGAP Education and Research Foundation answered the FDA's call for groups to submit a Letter of Intent (LOI) to the FDA requesting to host an externally-led Patient Focused Drug Development (PFDD) meeting. The FDA recently accepted and approved the LOI for SYNGAP1 submitted by Bridge the Gap – SYNGAP Education and Research Foundation. This meeting will give the SYNGAP1 community an opportunity to share personal insights that can be invaluable to the process of developing new treatments for the disease.
This groundbreaking initiative will culminate in a meeting featuring a series of facilitated panel discussions designed to provide the Food and Drug Administration (FDA) with caregiver perspectives on coping with the effects SYNGAP1. Following the successful model the FDA developed to host similar meetings, the half day meeting will focus primarily on a range of patient viewpoints on SYNGAP1; covering the symptoms and impacts to daily life that are most important to the families living with SYNGAP1, as well as their perspective on existing and future treatments. This input will help inform the FDA's decisions and oversight during future drug development and review of marketing applications for new drugs to treat SYNGAP1.
During the spring of 2020, the organization will begin to gather input from SYNGAP1 caregivers across the country and world through surveys and personal stories. At that time, information on how to participate will be made widely available throughout the international SYNGAP1 community.
Intellectual disability (ID) is a common disorder defined by the presence of significant limitations in both cognitive and adaptive behaviors with onset before the age of 18. ID is subdivided into syndromic intellectual disability, in which intellectual deficits and distinguishing morphologic, radiologic, or metabolic features are present, and non-syndromic intellectual disability (NSID), in which intellectual deficits appear without these physical abnormalities. Mutations in the SYNGAP1 gene are thought to be a relatively common cause of NSID. Patients with NSID, including those associated with SYNGAP1 mutation, typically exhibit moderate-to-severe ID with varying degrees of epilepsy and/or autism spectrum disorders (ASD); they may also have attention deficits, impulsivity, and/or mood disorders. Patients who have SYNGAP1-related NSID with epilepsy usually respond well to medications, yet some are refractory (difficult to control even with multiple drugs). SYNGAP1-related NSID is a sporadic condition caused mostly by de novo (spontaneous, non-inherited) mutations. The use of genomic sequencing has dramatically increased the capacity of physicians to identify these mutations.
About the FDA PFDD Initiative:
For more information about the Externally-Led FDA PFDD Initiative Meetings, Click HERE
About the Hosting Organization
Bridge the Gap – SYNGAP Education and Research Foundation (501(c) 3) is a non-profit organization whose mission is to serve, educate and fund research for families coping with the effects of SYNGAP1 mutations. The leading organization advocating and raising funds for research and treatments for SYNGAP1. Our international outreach for SYNGAP1 children gathers critical information, which is needed to drive research towards more immediate therapeutic solutions. Our mission is to improve the quality of life for people affected by SYNGAP1, provide family support, accelerate research and raise awareness so that every family and every child with SYNGAP1 can join us on the journey to a cure.
To help Sponsor the International SYNGAP1 Conference and SYNGAP1 PFDD External Meeting find out more information HERE