Exciting News! Ultragenyx's UX111 accelerated approval application is back under review at the U.S. Food and Drug Administration!
We are hopeful that FDA’s review will enable this transformative treatment to reach children living with Sanfilippo syndrome type A as soon as possible.
Our entire community is anxiously awaiting a first-ever approved drug for any type of Sanfilippo!
A promising gene therapy for Sanfilippo syndrome—a relentless disease that robs children of speech, mobility, and independence—is stalled by FDA delays. Trials show UX111 can slow or preserve these abilities, offering hope where no treatments exist.
Yet despite strong data, approval was rejected over minor manufacturing issues, delaying access for at least 6–12 months. For children with Sanfilippo, every month means irreversible loss.
Our kids are losing their voices, so we need yours to help by contacting HHS Secretary Kennedy and FDA Commissioner Makary and ensure:
Swift review of resubmissions for rare disease drugs, especially for Sanfilippo and Barth.
Engagement with sponsors to resolve minor issues before rejecting applications.
The FDA holds direct meetings with patient communities impacted by recent delays, including Sanfilippo and Barth.
The FDA makes full use of existing regulatory flexibility when the science is strong and safety risks are minimal.
To help, pick one or all three of the options below. This isn’t just about Sanfilippo. Other rare disease patients are facing similar delays, and we need reform.
Add your information to this template letter and it will automatically be sent to your members of Congress.
Calls are even more important! Staffers track every single call that comes in to show members of Congress what is important. Staffers will simply be listening and taking notes, so don't stress!
Call and request to meet with your Congress member using the talking points we've prepared. You don't have to be an expert! Meetings can be in person or virtual.
This letter is to FDA Commissioner Makary and others at the FDA. It is intended to draw attention to our rare disease community's urgent need for approved treatments. We need your help to send a message that as a rare disease community, we are watching, and we are strong together....
Share posts from Sanfilippo families sharing their stories or engage with them on social media to amplify the message.
Social media platforms: BlueSky, Facebook, Instagram, TikTok, TruthSocial, Twitter/X, YouTube, etc.
Tag:
• X: @US_FDA @MartyMakary @VPrasadMDMPH + your Congress reps
• Instagram: @fda @martymakary @drmakaryFDA @seckennedy
@robertfkennedyjr @karolineleavitt
+ your local Congresspeople and media
Hashtags: #ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary
Sanfilippo Syndrome is a terminal, degenerative disease that causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, dementia, and then die usually in their teen years. It is often referred to as being like a late-stage Alzheimer’s, but in children.
Sanfilippo is a rare genetic disease. Parents are unknowingly carriers of a genetic mutation that causes the children to have this condition. It is estimated that 1 in 70,000 births result in Sanfilippo. This equates to many thousands around the world suffering from this disease. Because it is rare, it is often left to parent-led organizations to spread awareness and fund research.
