uniQure Submits BLA for Gene Therapy in HD, AAN, AHS Issue Updated Guideline on Migraine Prevention, CTE Prevalence, Brain Donation Findings in Former NFL Players

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Beginning with FDA news, uniQure has submitted a Biologics License Application (BLA) to the FDA for accelerated approval of ifezuntirgeneinilparvovec (AMT-130), an investigational gene therapy for Huntington disease. The company also submitted a Marketing Authorisation Application (MAA) for the therapy to the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA).¹

uniQure has requested priority review for the BLA, which, if granted, would shorten the FDA review cycle to 6 months following the agency's 60-day BLA filing review period. Ifezuntirgeneinilparvovec is the first investigational Huntington disease therapy to receive both breakthrough therapy and regenerative medicine advanced therapy (RMAT) designations from the FDA, and it also holds a fast track designation.¹

"The submission of licensing applications for ifezuntirgeneinilparvovec represents an important milestone for the Huntington disease community," Matt Kapusta, chief executive officer of uniQure, said in a statement.¹ Kapusta said the company is grateful to the FDA for its leadership in advancing regulatory science to meet the urgency of the disease and to the MHRA for its commitment to advancing rare disease treatments in the UK, adding that uniQure looks forward to working with both agencies as the applications progress.¹

Switching gears, the American Academy of Neurology (AAN) and American Headache Society (AHS) have issued an updated practice guideline on pharmacologic migraine prevention in adults, replacing recommendations last updated in 2012.^1,2 Developed by a multidisciplinary panel based on a systematic review of evidence through June 2024,³ the guideline provides graded recommendations on when to start preventive therapy, how to select among available medications, and how to manage treatment in specific patient populations.

“There are a variety of effective preventive medications that work in different ways, including newer classes of medications that have been released in the past several years,” Tamara Pringsheim, MD, MSc, professor in the Department of Clinical Neurosciences, Psychiatry, Pediatrics and Community Health Sciences at the University of Calgary, and a fellow of the AAN, said in a statement.² “For people experiencing frequent migraine attacks or attacks that affect the ability to function normally, this guideline can help clinicians determine which preventive medications may be able to help.”

The guideline recommends preventive therapy for patients with 4 or more migraine or moderate to severe headache days per month, or substantial migraine-related disability, with treatment selection based on efficacy, tolerability, safety, cost, comorbidities, and patient preferences. Clinicians should consider factors such as pregnancy, age, comorbidities, and medication overuse, while monitoring response and adverse effects and adjusting or switching therapy when needed. Most preventives should be assessed after 8 to 12 weeks, with onabotulinumtoxinA assessed after 24 weeks.

A new retrospective population-based cohort study is adding to the growing body of research examining chronic traumatic encephalopathy (CTE) among former NFL players, while raising important questions about how the disease can be studied and its prevalence estimated. The research examined former players who died between 2008 and 2021, using postmortem neuropathological assessments alongside population-level mortality data.⁵

CTE remains a complex area of research, in part because definitive diagnosis currently relies on neuropathological examination after death. As interest continues to grow around the potential long-term neurological consequences of repetitive head impacts, researchers have increasingly sought to better understand the relationship between CTE pathology, clinical symptoms, and neurodegenerative conditions such as dementia.

In the first part of this 2-part Q&A with NeurologyLive, Thayne Munce, PhD, a member of the leadership council at the Wu Tsai Human Performance Alliance, discusses the study's approach to examining CTE in former NFL players, the broader significance of its findings, and the challenges involved in interpreting brain-donation research. Munce, who is also the site director of the Alliance at the University of Kentucky, also offered perspective on what the study may contribute to the ongoing understanding of CTE and its potential clinical manifestations.

To read the full pieces and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. uniQure N.V. uniQure Announces Submission of Biologics License Application for IfezuntirgeneInilparvovec (AMT-130) in Huntington's Disease. News release. Published September 2, 2026. Accessed September 2, 2026. https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-submission-biologics-license-application
2. Potrebic S, Tanveer S, Becker WJ, et al. Pharmacologic Treatment for Migraine Prevention in Adults Practice Guideline Recommendations: Report of the AAN Guidelines Subcommittee and the American Headache Society. Neurology. 2026;107(7):e214881. doi:10.1212/WNL.0000000000214881
3. AAN and AHS issue updated guideline on migraine prevention medications for adults. News release. American Academy of Neurology. August 31, 2026. Accessed September 1, 2026. https://www.aan.com/PressRoom/Home/PressRelease/5361
4. Pringsheim T, Smith DB, Tanveer S, et al. Systematic Review of Pharmacologic Treatment for Migraine Prevention in Adults: Report of the AAN Guidelines Subcommittee and the American Headache Society. Neurology. 2026;107(7):e218112. doi:10.1212/WNL.0000000000218112
5. Daneshvar D, Nowinski C, Abdolmohammadi B, et al. Prevalence of chronic traumatic encephalopathy at death in National Football League players: retrospective population based cohort study, 2008-21. BMJ. 2026;394. doi: 10.1136/bmj-2026-100418