‘Ready to fight the fight’: Huntington’s disease community delivers petitions to FDA seeking approval of uniQure gene therapy - By Gene Veritas

On January 22, Huntington’s disease organizations delivered two petitions to the FDA, demanding that the agency reverse its rollback on uniQure’s application for its HD gene therapy. In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, reflecting what critics saw as growing dysfunction under the Trump administration, the FDA abruptly switched gears regarding its agreed-to plan for considering AMT-130. HD advocates quickly organized two online petitions, garnering 48,000-plus signatures. “We can’t delay any longer,” Lauren Holder, a Help4HD International advocate and (like me) an HD gene carrier desperate for a therapy like AMT-130, said in a Facebook video at the FDA. “It’s important that they hear us.” Holder said that she often hears from HD-affected individuals that “‘I don’t want to be dying from HD; I want to be living with HD.’ These gene therapies and gene-modifying drugs are what get us to that point.” In a final, intense push for signatures, Holder on her January 16 podcast interviewed petition organizer Jeremy Renz (pictured here) whose wife tested positive for HD. “There’s more young people that are testing,” Renz told Holder. “There’s more young people who are at risk that are getting ramped up and ready to fight the fight.” Crucially, the high priority meeting that uniQure will soon hold with the FDA will include a representative of the community. Holder and Renz saluted how the HD community had united to work hard on AMT-130 advocacy. Read more in my latest article [Gene Veritas}. At Risk for Huntington's Disease: ‘Ready to fight the fight’: Huntington’s disease community delivers petitions to FDA seeking approval of uniQure gene therapy