The FDA has accepted Teva Pharmaceuticals’ new drug application 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.
Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer at Teva, said the FDA’s acceptance of the ecopipam application and grant of Priority Review is encouraging. 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). A subsequent open-label extension followed 121 pediatric participants for up to 12 months and reported sustained tic control.
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). This designation indicates a 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 said the FDA Fast Track designation for remlifanserin is a notable development. 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.
Related: Patients left at risk by poor communication standards
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.
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.
As the development of these treatments continues, the focus on ecopipam and remlifanserin highlights the ongoing search for more targeted therapies that can address specific symptoms or pathways involved in these conditions, such as neurodegenerative disease research.
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).