FDA issues priority vouchers for psychedelics, approves hearing-loss gene therapy under voucher pilot
The US Food and Drug Administration rolled out a series of moves this week, issuing priority vouchers to developers of psychedelic medications, approving a first-of-its-kind gene therapy under a voucher pilot, and reporting progress in cutting animal testing.
The agency said it issued vouchers under the Commissioner’s National Priority Voucher Program to three companies studying serotonin-2A agonists and related products, a class of psychedelic medications. The projects include psilocybin for treatment-resistant depression and major depressive disorder, and methylone for post-traumatic stress disorder.
FDA also said that following receipt of an investigational new drug submission, it is allowing an early-phase clinical study of noribogaine hydrochloride to treat alcohol use disorder. The agency noted it plans to issue a final guidance on the topic soon as a response to an executive order to accelerate access to treatments for patients with serious mental illness.
Separately, Regeneron Pharmaceuticals’ Otarmeni (lunsotogene parvec-cwha) was approved under the voucher pilot program as the first dual adeno-associated virus vector-based gene therapy for patients with severe-to-profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene.
FDA said the therapy was approved 61 days after the company filed a biologics license application and is the sixth product approved under the voucher program. FDA announced it has rescheduled a public meeting to discuss the voucher pilot program. The hybrid meeting, originally set for June 12, will now be held on June 4.
Marking one year of its initiative to reduce animal testing, the agency said it has achieved its key objectives, including issuing new draft guidance and other updated guidance, launching the first artificial intelligence-based drug development tool, and creating a database that outlines when alternative methods are acceptable.
FDA will host its annual hybrid Regulatory Education for Industry (REdI) Conference on May 19–20. The agency said the meeting is intended to give stakeholders a chance to learn from its experts about how it regulates medical products, provide a basic foundation on regulatory requirements, and offer updates on current activities.
The Center for Devices and Radiological Health published the latest meeting minutes with non-industry stakeholders—including the American Academy of Pediatrics, the National Organization for Rare Disorders, the Personalized Medicine Coalition, and Public Citizen—as negotiations continue on the next Medical Device User Fee Amendments (MDUFA VI) program.
According to the minutes, stakeholders requested more information on IT tools, the Total Product Lifecycle Advisory Program, digital health, continuous process improvements for premarket review, international fees, trigger reform, and the use of real-world evidence, among other topics.
CDRH also classified several products as class II devices with special controls. The list includes digital therapy devices for amblyopia, Alzheimer’s disease pathology assessment tests, setmelanotide eligibility gene variant detection systems, and devices for sleep apnea testing based on mandibular movement.
In addition, FDA published its congressionally mandated annual FY 2024 report on the status of postmarketing requirements and postmarketing commitments for approved drug and biological products, detailing studies and clinical trials that applicants are required to conduct or have agreed to conduct.
Next on the calendar: the voucher pilot program meeting on June 4 and the REdI Conference on May 19–20, with the agency also signaling that final guidance on psychedelic-drug development is forthcoming.