FDA Issues Complete Response Letter for Sydnexis’ SYD-101 Pediatric Myopia Drug Application

The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Sydnexis Inc. regarding its New Drug Application (NDA) for SYD-101, a 0.01% low-dose atropine formulation developed to slow the progression of myopia in children.

Background on SYD-101 and STAR Trial

SYD-101 is a proprietary formulation of low-dose atropine optimized for improved ocular delivery and room-temperature stability. The NDA was supported by results from the Phase 3 Study of Atropine for the Reduction of Myopia Progression (STAR) trial, one of the largest global studies conducted in pediatric myopia to date. The trial enrolled over 800 children aged 3 to 14 and followed them for three years.

According to Sydnexis, the STAR trial achieved its primary efficacy endpoint, which was the proportion of children with confirmed myopia progression of -0.75 diopters (D), a benchmark recommended by the FDA. Additionally, key secondary outcomes, including annual progression rate, showed statistically significant improvements at 12, 24, and 36 months.

In a subgroup of children identified as fast progressors (defined as progressing more than -0.5 D per year), SYD-101 demonstrated a reduction in myopia progression exceeding 50% after 36 months.

FDA's Complete Response Letter

In its CRL, the FDA acknowledged that the primary efficacy endpoint had been met. However, the agency concluded that the current evidence did not demonstrate sufficient effectiveness of low-dose atropine for treating pediatric myopia. The letter did not cite any concerns related to safety or product quality, according to the company.

Sydnexis Response

"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, CEO of Sydnexis. “Our goal is to provide eye care professionals in the US with the first FDA-approved option to help slow the progression of pediatric myopia, rather than continued reliance on compounded formulations of atropine."

Sternberg also reaffirmed the company's confidence in the STAR trial data and SYD-101’s potential to meet a critical need in pediatric eye care, particularly given the increasing prevalence of childhood myopia.