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FDA accepts fenebrutinib filing for relapsing and primary progressive MS; hearing aids miss CHOICE end point; Congress passes ACT for ALS renewal
THE SHORT READ
Genentech said Sept. 29 that the Food and Drug Administration (FDA) accepted its application for fenebrutinib in relapsing and primary progressive multiple sclerosis (MS) under priority review. In JAMA Neurology, the CHOICE trial found that hearing aids did not significantly reduce conversion from mild cognitive impairment (MCI) to dementia-level impairment.
Congress sent the ACT for ALS reauthorization to the president, and a phase 3 trial in Niemann-Pick disease type C missed its primary end point.
THE DETAILS
- FDA Sept 29, 2026Genentech said Sept. 29 that the Food and Drug Administration (FDA) accepted, under priority review, its new drug application for fenebrutinib, an investigational oral, central nervous system-penetrant, noncovalent Bruton's tyrosine kinase (BTK) inhibitor. The company described it as the first BTK inhibitor to receive FDA filing acceptance in both relapsing and primary progressive MS. In FENhance 1 and 2, fenebrutinib reduced the annualized relapse rate by 51.1% and 58.5% versus teriflunomide over 96 weeks; in FENtrepid, it met its noninferiority primary end point versus ocrelizumab (Ocrevus) on disability progression (hazard ratio 0.88; 95% CI 0.75 to 1.03), the company said.Sources: Genentech, Genentech press releases
- Research Sept 28, 2026In the open-label CHOICE randomized trial, published Sept. 28 in JAMA Neurology, 703 adults in Shanghai with MCI and moderate to severe hearing loss received a hearing aid intervention or hearing care education. Over 24 months, conversion to dementia-level impairment (a Clinical Dementia Rating global score of 1 or more) was 3.09% with hearing aids versus 4.74% with education, not a significant difference (relative risk 0.59; 95% CI 0.25 to 1.38; P = .23). In a prespecified secondary analysis, cognitive improvement occurred in 15.34% of the hearing aid group versus 2.36% of the education group.
- Policy Sept 28, 2026The Senate approved the Accelerating Access to Critical Therapies (ACT) for ALS Reauthorization Act of 2026 (H.R. 8205) after House passage, sending it to the president, the ALS Network said Sept. 28. The bill extends key ACT for ALS programs, which support research and expanded access to investigational therapies, through 2031; the current authorization was scheduled to expire Sept. 30. According to NeurologyLive, it adds review requirements for grant renewals tied to investigational drugs and directs the Food and Drug Administration to develop or update an action plan for rare neurodegenerative diseases.
- Research Sept 30, 2026In topline results released Sept. 30, Rafael Holdings said intravenous Trappsol Cyclo did not meet the primary end point of the phase 3 TransportNPC trial, the change in 4-domain NPC Clinical Severity Scale score at week 96. Among 94 patients aged 3 to 70 years, the least squares mean change was 0.46 points with Trappsol Cyclo and 1.28 points with placebo (P = .19). In a prespecified analysis of 78 patients on background miglustat, leucine or both, the changes were 0.46 and 1.57 points (P = .046); the company plans a new drug application in the fourth quarter of 2026.Sources: Rafael Holdings, NeurologyLive