Key Takeaways

• The FDA informed Sydnexis of its intent to convene an Advisory Committee meeting to discuss SYD-101, an investigational low-dose atropine therapy for pediatric progression myopia.
• In the phase 3 STAR trial, SYD-101 0.01% met its primary (P = 0.0226) and key secondary endpoint (P < 0.001) and was well tolerated with no unexpected atropine-related adverse events.
• The date of the Advisory Committee meeting has not yet been set.

Sydnexis announced that the FDA has informed the company of its intent to convene an Advisory Committee meeting to discuss SYD-101, an investigational low-dose atropine therapy for the treatment of pediatric progression myopia. While the date for the meeting has not been set, the FDA has indicated that the Advisory Committee will be asked to discuss various aspects of Sydnexis’ new drug application, including findings from the phase 3 STAR study. Sydnexis submitted a Formal Dispute Resolution Request to the FDA’s Office of Specialty Medicine after receiving a Complete Response Letter in 2025.

The phase 3 STAR trial evaluated a population of 847 children from 3 to 14 years of age at treatment initiation. Participants with myopia of -0.50 D to -6.00 D, with a mean baseline of -2.69 D, were enrolled across the United States and Europe and randomized (1:1:1) to vehicle (placebo) and SYD-101 0.01%. The primary efficacy endpoint was the proportion of patients with confirmed progression of -0.75 D, and a key secondary endpoint was annual progression rate. SYD-101 0.01% successfully met both the primary endpoint (P = 0.0226) and key secondary endpoint (P < 0.001) and was well tolerated with no unexpected atropine-related adverse events.

"We appreciate the FDA's decision to quickly convene an Advisory Committee meeting and have requested it include practicing pediatric ophthalmologists and optometrists, as they understand the long-term challenges facing patients and families every day," said Perry Sternberg, Chief Executive Officer of Sydnexis, in a press release. "For practicing physicians and those living with PPM, the need for an FDA-approved treatment option is not a theoretical question. We believe this meeting provides an important opportunity to have a robust, science-led discussion around the totality of evidence supporting SYD-101 and we look forward to hearing the perspectives of clinicians who treat PPM on a daily basis."

SYD-101 is currently approved in the European Union and United Kingdom, where it is licensed to Santen and marketed as Ryjunea.