FDA Accepts Ecopipam NDA for Tourette Syndrome, Remlifanserin Receives Fast Track Designation for AD Psychosis, Alterity Reaches FDA Alignment on ATH434

WATCH TIME: 6 minutes | Captions are auto-generated and may contain errors.

Below is a transcript of the video.

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

Starting with FDA news, the administration has accepted Teva Pharmaceuticals' new drug application (NDA) for ecopipam, a first-in-class selective dopamine D1 receptor antagonist under investigation for pediatric patients with Tourette syndrome (TS), and granted the submission priority review. The company noted that the agency set a target action date under the Prescription Drug User Fee Act (PDUFA) for late in the first quarter of 2027.¹

Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer at Teva, told NeurologyLive^® this, “We are encouraged by the FDA's acceptance of the ecopipam application and grant of Priority Review. Tourette syndrome is an area where innovation has been limited for far too long, with patients and clinicians often relying on treatments developed for other conditions,”

The NDA application is supported by data from the phase 2b and phase 3 D1AMOND clinical program. In the 12-week, randomized, double-blind, placebo-controlled phase 2b trial, 153 pediatric participants across 68 sites in North America and Europe were randomized to ecopipam or placebo. Findings showed that those receiving ecopipam had a statistically significant and clinically meaningful reduction in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) versus placebo at week 12 (P = .01).^1,2 A subsequent open-label extension followed 121 pediatric participants for up to 12 months and reported sustained tic control.

“The Phase 2b and Phase 3 data supporting this application demonstrated meaningful and durable reductions in tic severity with a favorable tolerability profile, giving us confidence in ecopipam's potential as a differentiated treatment option,” Hughes added. “If approved, we believe ecopipam's novel D1 receptor mechanism could provide clinicians with an important new option to help children and adolescents better manage their symptoms and the impact those symptoms can have on daily life."

Continuing with FDA news, the FDA has granted Fast Track designation to remlifanserin (ACP-204; Acadia Pharmaceuticals), an investigational selective serotonin 5-HT2A receptor inverse agonist, for the treatment of hallucinations and delusions associated with Alzheimer disease psychosis (ADP).²

“FDA Fast Track designation for remlifanserin underscores the significant unmet need for new treatment options for people living with Alzheimer disease psychosis, a serious condition for which there are currently no FDA-approved therapies,” Catherine Owen Adams, chief executive officer of Acadia, said in a statement.² “This is an important milestone for the remlifanserin program, and we look forward to sharing upcoming topline data from the RADIANT Phase 2 trial as we continue to advance remlifanserin as a potential new treatment option for patients and families impacted by this devastating condition.”

The regulatory milestone comes as Acadia advances remlifanserin through the RADIANT development program. Enrollment in the phase 2 portion of the program has been completed, with topline results expected between September and October 2026. Screening and enrollment are also underway in several phase 3 studies as part of RADIANT’s design.

In early June, Alterity Therapeutics announced that it had achieved alignment with the FDA following a successful End-of-Phase 2 meeting for ATH434, its investigational oral iron redistribution agent in development for multiple system atrophy (MSA). The FDA agreed on the key elements of the proposed Phase 3 design, including the study population, the 50 mg twice-daily dosing regimen, a 12-month treatment duration, and the 11-item UMSARS Part 1 as the primary efficacy endpoint. The alignment clears the path for a pivotal registrational trial, with activities on track to initiate by year-end 2026.³

In a randomized, double-blind, placebo-controlled Phase 2 trial, ATH434 previously demonstrated a 46% relative slowing of disease progression on the UMSARS Part 1 compared with placebo with a favorable safety profile and neuroimaging findings consistent with reduced toxic iron accumulation in affected brain regions. The drug has also received Fast Track and Orphan Drug designations from the FDA, underscoring the significant unmet need in MSA, a rapidly progressive neurodegenerative disease where the median survival is approximately 7.5 years from symptom onset and no disease-modifying therapies are currently approved.

In a Q&A published by NeurologyLive, David Stamler, MD, Chief Executive Officer of Alterity, discusses what FDA alignment at the End-of-Phase 2 stage means for the ATH434 development program, why UMSARS Part I was selected as the primary endpoint, how the Phase 2 findings shaped the Phase 3 design, and what a successful pivotal trial could ultimately mean for the up to 50,000 individuals living with MSA in the United States.

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. U.S. FDA Accepts Teva's New Drug Application (NDA) and Grants Priority Review for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Patients with Tourette Syndrome. News release. Teva Pharmaceuticals. August 19, 2026. Accessed August 19, 2026. https://www.tevapharm.com/news-and-media/latest-news/u.s.-fda-accepts-tevas-new-drug-application-nda-and-grants-priority-review-for-ecopipam-a-first-in-cl
2. Acadia Pharmaceuticals Receives FDA Fast Track Designation for Remlifanserin in Alzheimer's Disease Psychosis. Acadia Pharmaceuticals. News Release. July 20, 2026. Accessed August 17, 2026. https://www.businesswire.com/news/home/20260720458854/en/Acadia-Pharmaceuticals-Receives-FDA-Fast-Track-Designation-for-Remlifanserin-in-Alzheimers-Disease-Psychosis
3. Alterity Therapeutics achieves alignment with U.S. FDA on pivotal phase 3 program for ATH434 in multiple system atrophy. GlobeNewswire. June 9, 2026. Accessed August 17, 2026. https://www.globenewswire.com/news-release/2026/06/09/3308715/0/en/alterity-therapeutics-achieves-alignment-with-u-s-fda-on-pivotal-phase-3-program-for-ath434-in-multiple-system-atrophy.html