Regulatory
Center for Biologics Evaluation and Research Director Vinay Prasad will exit the FDA for the second time after a controversial run that saw the oncologist butt heads with colleagues and ruffle feathers in biopharma after presiding over the rejection of several rare disease therapies.
UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.
Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.
The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024.
Having targeted Hims & Hers last year, the FDA has issued warnings to more telehealth companies over their promotion of GLP-1 drugs used to treat diabetes and obesity. In one case, compounded products were linked with multiple ER visits.
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.
While an anonymous source tied the closure to shortcomings in the FDA’s new pathway, a spokesperson for the Department of Health and Human Services pushed back on the suggestion.
Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies.
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking.
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