SPRINGFIELD — Today, Congresswoman Nikki Budzinski (IL-13) called on the Food and Drug Administration (FDA) to consider urgent approval of a potentially life-saving treatment for four-year-old Lottie Milburn, a Springfield child diagnosed with Sanfilippo Syndrome — a rare and fatal genetic disorder that affects the brain and spinal cord.
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In a letter to the agency, Budzinski encouraged the FDA to consider expediting its review of the resubmitted Biologics License Application (BLA) for Ultragenyx’s UX111 gene therapy. The treatment has shown promising results in clinical trials and offers new hope for families affected by Sanfilippo Syndrome.
“Historically, BLA reviews can take up to six months — time that many families simply don’t have,” Budzinski wrote. “Acting swiftly to approve UX111 could save the lives of thousands of children who are running out of time.”
She continued, “As such, I encourage the FDA to review the resubmitted BLA for Ultragenyx’s UX111 treatment as soon as possible and pledge to work with the rare disease community to ensure no child is denied access to lifesaving treatment.”
To read more about Lottie’s story, click HERE. To view the letter to the FDA, click HERE.
