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By Jasmine Laws
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More than 100 mothers and advocates affected by a type of rare disease gathered outside the Food and Drug Administration (FDA) on Wednesday to stage a funeral to "mourn the futures" of their children, following the agency's rejections of various rare disease treatments.
The community, affected by a group of rare metabolic disorders known as mucopolysaccharidoses (MPS), dressed in black, held signs and carried a coffin outside the FDA's headquarters in a peaceful protest, demanding that treatments become available for MPS conditions such as Sanfilippo syndrome, Hurler syndrome and Hunter syndrome.
There are no approved treatments for Sanfilippo syndrome and no brain-penetrant therapies approved for Hurler syndrome and Hunter syndrome, even though all three are considered life-threatening.
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Kim Stephens, the executive director of the Dr. Joseph Muenzer MPS Research and Treatment Center at the University of North Carolina at Chapel Hill, whose son Cole has Hunter syndrome, told Newsweek that the protest was "important for our families to stand up for their children and their futures. The FDA won't approve safe and effective drugs, and their children will die because of it."
"The FDA needs to see the faces of the children they are condemning to death. We want the president to see them and help us," she added.
The FDA previously told Newsweek, "The FDA is diligently committed to delivering cures and treatments to families in need in the safest way possible."
Why It Matters
The FDA has come under increasing pressure in recent months over its denials of certain rare disease treatments. Rare disease advocates have said the agency's rejections show signs of inconsistencies around regulatory requirements, slowing down the process of getting treatments across the line for suffering Americans, while the FDA has maintained that approvals in general have not gone down.
Through clinical trials, some children have come to rely on the drugs being denied FDA approval, and without the agency's approval, they could lose access to said drugs, leaving their families concerned for their futures.
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What To Know
The FDA will send out a complete response letter if it decides the drug in question cannot be approved in its present form. This typically means the drug is rejected or its approval is delayed until additional investigation into its safety and effectiveness as a treatment is completed.
Kim Higbee, a board member of the Hope for PDCD Foundation, an organization supporting those with pyruvate dehydrogenase complex deficiency, previously told Newsweek that since the start of January 2025, the rare disease community saw the FDA send 23 complete response letters regarding rare disease drugs.
A Department of Health and Human Services spokesperson previously told Newsweek that the number of FDA approvals and rejections under the Trump administration were "consistent with historical data over the last decade."
On July 11, 2025, Ultragenyx Pharmaceutical announced that the FDA had issued a complete response letter for its gene therapy UX111, designed for the treatment of Sanfilippo syndrome type A.
On January 28, drug manufacturer Regenxbio announced that the FDA had placed a "clinical hold" on its gene therapy RGX-111, a potential treatment of Hurler syndrome, after preliminary analysis found a brain tumor in a 5-year-old participant treated four years prior.
The same manufacturer also received a complete response letter for its gene therapy RGX-121, a potential treatment for Hunter syndrome, on February 9.
Gathering outside the FDA on Wednesday, the rare disease community called on the agency to implement regulatory flexibility for ultrarare diseases with no approved treatments and accelerate pathways and biomarker-based end points for MPS conditions.
The group also called on Congress to oversee the FDA's decision-making, while including meaningful patient and family engagement throughout drug development and review.
Mark Dant, the volunteer executive director of the Ryan Foundation, told Newsweek that part of the reason these drugs have been denied is because of the FDA's "dislike" of the accelerated approval pathway.
"This pathway, codified by Congress, is the only way to keep the science that can treat our children moving forward to approval, and yet because of personal dislikes by Director Prasad, our children continue to suffer and die," he said. "We can no longer stand by and wait to plan their funerals."
"For decades we waited for science to find our tomorrows," he added. "Now, it has, and bureaucrats within the agency we pay for are keeping those treatments from our children. We know they are there. ... We just cannot reach them."
Stephens' son Cole, a 15-year-old with Hunter syndrome, is one of the children affected by the FDA's lack of approval of Regenxbio's gene therapy.
He isn't able to speak, but he can go for walks, feed himself and breathe without assistance. He said his last word at age 9, which was "Mommy."
"This is his last chance, and this is true for so many others, even outside of MPS," Stephens said. "Our MPS community has lost 54 people since July."
Stephens also told Newsweek that the MPS community has sent over 10 letters to FDA Commissioner Marty Makary asking for a meeting. "There is no answer," she said. "We received a form letter. I even put a request in Makary's hand in a bright orange envelope. No answer."
"We heard today that a senior leadership official at the FDA told them at the FDA to not go out and engage with us because they didn't want to reward bad behavior," she said, adding that the community would "keep speaking up."
When approached for comment in regard to these claims, the FDA told Newsweek it had sent a response earlier this year.
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What People Are Saying
Mark Dant, the volunteer executive director of the Ryan Foundation, said in a news release: "For over 30 years, we have traveled to the Hill and talked to our legislators. We meet with a staff member, take a picture, and nothing is done. We cannot continue to do this while children are dying."
Terri Klein, the president and CEO of the National MPS Society, said: "For families living with MPS and ML, every day without access to treatment is a day of irreversible damage. The science is there. The urgency is undeniable. It is time for decision-makers to match our determination with the regulatory courage these children deserve. I carry this fight personally; my own daughter is waiting for treatments that may never reach her if the FDA's regulatory uncertainty continues to cast a shadow over the breakthrough science that could change her life."
Kristin McKay, the president and executive director of Project Alive, said: "Every delay means children losing skills, losing health, and losing a future that science is finally making possible. We are asking Congress to hold the FDA accountable—and to keep pace with our children."
What Happens Next
The FDA's decisions around some of these rare disease treatments could be subject to review after Republican Senator Ron Johnson of Wisconsin said last week he would launch an investigation into the FDA over its denials.
Update 3/20/26, 4:28 a.m. ET: This article has been updated with response from the FDA.
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