SYD-101 (Sydnexis), a first-of-its-kind eye drop specifically developed to slow pediatric progressive myopia (PPM) progression, is still navigating major regulatory obstacles before it can become available to US patients.
In a complete response letter (CRL) to Sydnexis regarding its New Drug Application (NDA) for SYD-101, the U.S. Food and Drug Administration (FDA) acknowledged that the company’s Phase 3 trial met its primary efficacy endpoint but stated that the drug lacked sufficient evidence to support the effectiveness of low-dose atropine for myopia progression in children. SYD-101 is a proprietary 0.01% atropine eyedrop intended to slow the progression of pediatric progressive myopia (PPM) in children. The CRL raised no concerns related to safety or product quality.
“Despite positive findings, the FDA’s CRL emphasized that the data did not convincingly demonstrate the clinical effectiveness of low-dose atropine for all children in the studied population.”
SYD-101 is the only formulation of its kind developed specifically for pediatric myopia. Its benefits include:
- Enhanced ocular tissue permeability
- Near-neutral pH
- Room-temperature stability up to 3 years.
About the Trial Results
The NDA for SYD-101 was backed by the results of the Phase 3 STAR (Study of Atropine for the Reduction of Myopia Progression) trial, which enrolled more than 800 children aged 3 to 14 and is the largest global clinical program completed to date in pediatric myopia, the company says. The study achieved its primary efficacy endpoint, defined as the proportion of patients with confirmed myopia progression of -0.75 diopters (D), a measure encouraged by the FDA. Secondary endpoints, including annual progression rates at 12, 24, and 36 months, also reached statistical significance. Notably, a subgroup of “fast progressors” (more than -0.5D per year) experienced a reduction in myopia progression of more than 50% with SYD-101 therapy over 36 months.
Responses
Despite these positive findings, the FDA’s CRL emphasized that the data did not convincingly demonstrate the clinical effectiveness of low-dose atropine for all children in the studied population.
“While we are surprised and disappointed with this decision, Sydnexis is committed to working with the FDA to address the items outlined in the CRL and determining the best path forward toward approval for SYD-101,” said Perry Sternberg, Chief Executive Officer of Sydnexis. Mr. Sternberg added that the company remains confident in its data and SYD-101’s potential to treat PPM.
SYD-101 is currently approved and marketed in the European Union as Ryjunea.