12/04/2025
Update!
UPDATE on the Sanfilippo Type B Expanded Access opportunity and fundraising.
Just a month and a half ago, an opportunity arose to support the creation of a Sanfilippo Type B enzyme replacement drug for future use under an Expanded Access Program. Expanded access (EA) typically allows for broader access without the same restrictive inclusion criteria that are commonly seen in formal clinical trials. Timing of treatment is very important in Sanfilippo, so the earlier an intervention can come, the better. This is an incredibly unique opportunity, and came with a first goal of $3.8 million cost and an extremely short deadline to be able to secure the EA drug manufacturing slot by December 1st.
Parents and families of children with Type B, communities, and others came together and rallied to fundraise for two Foundations to reach this goal in this short window. The outpouring of generosity has been incredible and the work of the community has been literally non-stop. Online fundraising, events, news media spots, large donations via wire transfers, awareness campaigns, billboards in multiple cities and locations … you name it, these families have done it, in under two months’ time!
We are thrilled to announce that this first goal of $3.8M was reached. Fundraising through Cure Sanfilippo Foundation has generated $3.5M in Expanded Access-directed funding. Additional funds have also been raised through our partners at the National MPS Society to meet this first goal.
The final goal for this project is another $1.7M to complete drug preparation in the first quarter 2026. To help reach this next goal, the Cure Sanfilippo Foundation Board has approved up to an additional $1.2M to go toward either of these two goals for this EA program as needed.
These significant funds will be directed specifically to, manufacturing of the drug, safety testing, and delivery of the drug to sites for the intended Expanded Access Program in the United States, which is anticipated to become available later in 2026. The nature of this program is that there will be a limited amount of drug available to a limited number of individuals, for a limited time (estimated one year). However, we are excited to bring hope in the form of treatment and timely access to those who will be able to participate in the Expanded Access Program and who otherwise would not be able to take part in a clinical trial or access treatment. Future decisions about which clinical sites are eventually approved to deliver the drug and which individuals are enrolled in the Expanded Access Program will be in the hands of the physicians, the company, and FDA.
During these past weeks, Cure Sanfilippo has been interacting directly with the company and carefully evaluating this opportunity with due diligence and is moving forward with legal contracting in good faith as details of the expanded access program, including its full scope and timing, continue to take shape. Cure Sanfilippo has also been collaborating with sites in the U.S. that have administered this enzyme replacement previously to learn from their experience, engaging with the medical community to better understand current capacity and future pathways, and gather questions so that our community can continue to prepare while the drug manufacturing is in process. Ensuring that our children are treated safely and timely and that the hard-fought funds raised for this project are put to their intended use is of utmost importance.
This Expanded Access program will be a first of its kind in Sanfilippo and we are thankful the company desires to honor the urgent need for children. And thankful to the families and supporters who answered the call…just amazing! We look forward to more information from the company detailing how this program will roll out, and we will keep you updated.
