Novel Dexmethylphenidate Formulation Under Review for ADHD

The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for CTx-1301 for the treatment of attention deficit/hyperactivity disorder (ADHD) in children and adults. 

CTx-1301 is a novel proprietary formulation of dexmethylphenidate, a stimulant that increases norepinephrine and dopamine activity in the brain. The product is designed to deliver 3 separate releases of medication at different times and in different ratios, resulting in a more rapid onset of action and a longer efficacy time than other extended-release stimulants.

The approval was based on a clinical trial package that included a phase 3 adult dose-optimization study (CTx-1301-022; ClinicalTrials.gov Identifier: NCT05631626) as well as a phase 3 pediatric fixed-dose study (CTx-1301-005 trial; ClinicalTrials.gov Identifier: NCT05286762). 

The adult dose-optimization study assessed the efficacy and safety of CTx-1301 in patients aged 18 to 55 years with ADHD in a laboratory classroom setting. After a 5-week dose optimization period, study participants (N=21) were randomly assigned to receive CTx-1301 or placebo. Findings showed treatment with CTx-1301 resulted in a clinically meaningful improvement in attention based on permanent product measure of performance score (primary endpoint) compared with placebo. A statistically significant change from baseline in Clinical Global Impression (CGI)-Severity score was also observed with CTx-1301 (P <.001). 

The pediatric fixed-dose study evaluated the efficacy and safety of CTx-1301 in pediatric patients aged 6 to 17 years with ADHD. Study participants (N=103) were randomly assigned to receive CTx-1301 (18.75mg, 25mg, 37.5mg) or placebo for 5 weeks. The primary endpoint was the mean change from baseline in ADHD Rating Scale 5 (ADHD-RS-5) score at week 5.

Findings showed CTx-1301 statistically significantly reduced ADHD symptoms, based on ADHD-RS-5 score, in all doses tested (P =.018 [18.75mg]; P =.011 [25mg]; P =.001 [37.5mg]) vs placebo. Additionally, dose-dependent improvements in CGI-Improvement and CGI-Severity scales were reported.

As for safety, no serious treatment-emergent adverse events were reported across all studies. The tolerability profile was also similar to that of other long-acting methylphenidate products. 

“FDA acceptance of our NDA marks a defining milestone for Cingulate,” said Cingulate Executive Chairman Jay Roberts. “CTx-1301 was engineered to address the practical shortcomings of today’s stimulant therapies, multiple daily dosing, midday rebound, and adherence challenges, through a single, once daily tablet.”

A Prescription Drug User Fee Act target date of May 31, 2026 has been set for the application.

This article originally appeared on MPR