Sydnexis Releases Additional Data for SYD-101 Following FDA Refusal
On 23 October, the FDA issued a complete response letter (CRL) to Sydnexis, preventing SYD-101 from entering the US market for paediatric progressive myopia (PPM). Despite the setback, Sydnexis disclosed further trial data demonstrating that SYD-101 met both primary and secondary endpoints.
FDA Response and Trial Background
Although the FDA acknowledged the study hit its primary endpoint, they concluded the data do not sufficiently support the efficacy of low-dose atropine in children with myopia. The application was based on results from the Phase III STAR trial (NCT03918915).
New Data Presented at AMCP Nexus 2025
Sydnexis shared updated results during a poster session at the Academy of Managed Care Pharmacy (AMCP) Nexus 2025 in National Harbor, Maryland. The data indicated that the greatest clinical benefit in spherical equivalent (SE) change was observed in younger patients, aged 3 to 12 years at the start of treatment.
- For the full cohort, SE changes after treatment were -0.71 for SYD-101 0.01% and -0.76 for SYD-101 0.03%, compared to -0.92 in the vehicle (placebo) group.
- Both doses showed nominal statistical significance in these younger patients.
- Additional data was presented on a subgroup of fast progressors.
"While the FDA acknowledged that the study met this primary endpoint, the letter stated that the data do not support the effectiveness of low-dose atropine in children with myopia."
These findings highlight a nuanced clinical effect of SYD-101 in specific patient groups despite regulatory challenges.
Author's summary: Sydnexis shared promising new data on SYD-101’s effectiveness in young children with progressive myopia shortly after the FDA blocked its US market approval, showing notable treatment effects in certain subgroups.