More than 100 marchers took to Capitol Hill on Wednesday after carrying a coffin to the FDA in a symbolic protest highlighting lives they say could be lost to regulatory delays affecting a group of rare metabolic disorders, according to a release shared with Fierce.
The demonstration comes as controversy around the FDA’s rare disease decisions continues to escalate following uniQure’s heated back-and-forth and several unexpected rejections.
Today, the advocates, families and caregivers turned out to demand action for patients with mucopolysaccharidoses (MPS), conditions that are caused by a deficiency of enzymes needed to break down sugar changes in cells. Currently, no approved treatments exist for MPS III—also known as Sanfilippo syndrome—while no brain-penetrant therapies are marketed for MPS I, also called Hurler syndrome, or MPS II, also called Hunter syndrome.
The marchers dressed in black and staged a funeral outside the FDA, demanding that the regulator and the Congress “stop playing politics with treatments that science has proven safe and effective,” according to the release from the National MPS Society.
The nonprofit has previously spoken out about the agency’s rebuff of Regenxbio’s gene therapy designed to treat Hunter syndrome. The therapy, called RGX-121, was rejected in early February, with the FDA citing concerns about the ability to properly define a patient population, the use of a natural history control arm and the use of a biomarker as a surrogate endpoint in the sole trial submitted.
Three drugs developed for Hunter syndrome have been denied or delayed by the FDA, actions that the advocates believe are because of the “agency’s ideological opposition to accelerated approval pathways and the use of biomarkers as clinical endpoints.”
“The FDA is diligently committed to delivering cures and treatments to families in need in the safest way possible,” a spokesperson for the Department of Health and Human Services told Fierce. “That means requiring clear evidence of clinical benefit for the intended patient populations. Companies must demonstrate that benefit through strong data from a well-controlled and adequately-run clinical trial.”
“The American people voted for an FDA that works for them—not just the sponsors—and the agency is delivering by refusing to rubber-stamp approvals,” the spokesperson added. “Under Commissioner [Marty] Makary’s leadership, FDA approvals are at their peak, driven by the agency’s commitment to first-class science.”
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“It is time for decision-makers to match our determination with the regulatory courage these children deserve,” National MPS Society President and CEO Terri Klein said in a March 18 statement.
Alongside the National MPS Society, the “public mourning” was organized by Project Alive, The Ryan Foundation and The Kennedy Ladd Foundation.
“For over thirty 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,” Mark Dant, volunteer executive director of The Ryan Foundation, said in the release. “We cannot continue to do this while children are dying.”
The demonstrators are demanding four things: immediate regulatory flexibility for ultrarare diseases with no approved treatment; FDA willingness to use accelerated approval pathways and biomarker-based endpoints for MPS conditions; meaningful patient and family engagement throughout the process; and congressional oversight of FDA decision-making in rare diseases without treatments.
As the rare disease community continues to speak out against certain FDA actions, questions have been raised about the ethics of Congress’ involvement in regulatory decision-making, given its inherently political nature.
Last week, after U.S. Sen. Ron Johnson, R-Wis., launched an investigation into recent approval rejections for rare disease candidates, the University of Pennsylvania’s associate professor of medical ethics and law Holly Fernandez Lynch wrote: “The White House and Congress (and the FDA commissioner) all have a legitimate role in shaping FDA policy around drug approvals. But they should not intervene in decisions about specific drugs or we risk not knowing whether those decisions were driven by evidence or politics.”
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“No one should be playing politics with our children’s lives,” Kim Stephens, mother of Cole, a 15-year-old with Hunter syndrome, said in the National MPS Society release.
“The science is sound and safe,” Stephens added. “Every day the FDA delays is a day a child loses—skills, health, time. We are out of time.”
