FDA to Convene Advisory Committee to Review Sydnexis's SYD-101 for Pediatric Progressive Myopia
核心洞察
The FDA plans to hold an advisory committee meeting to review SYD-101, a low-dose atropine 0.01% formulation for pediatric progressive myopia (搜索), following a prior complete response letter rejecting its NDA.
SYD-101 met its phase 3 STAR study primary and secondary endpoints, including significant reduction in myopia progression at 36 months (p = 0.0226), yet the FDA questioned the durability and robustness of the treatment effect.
Sydnexis has requested that practicing pediatric ophthalmologists and optometrists be included on the advisory committee to ensure real-world clinical perspectives are represented.
The FDA has informed Sydnexis, Inc. of its plans to convene an advisory committee to review SYD-101, an investigational low-dose atropine 0.01% formulation intended for the treatment of pediatric progressive myopia (搜索) (PPM). The announcement comes nearly nine months after the agency issued a complete response letter (CRL) declining to approve the eye drop's new drug application (NDA), despite the pivotal phase 3 STAR study meeting both its primary and secondary efficacy endpoints.
No date has yet been set for the advisory committee meeting. According to Sydnexis, the FDA indicated that the committee will be asked to discuss various aspects of the company's application, including findings from the phase 3 STAR study.
SYD-101: A Novel Formulation with Distinct Properties
SYD-101 is a patented, proprietary 0.01% atropine formulation designed to slow myopia progression in pediatric patients aged 3 to 14. The formulation incorporates several features intended to optimize clinical performance, including enhanced ocular tissue permeability demonstrated in preclinical animal studies, a stable shelf-life of up to three years at room temperature, and a near-neutral pH that may support a favorable ocular safety and comfort profile.
If approved, SYD-101 would become the first and only FDA-approved pharmaceutical treatment for progressive myopia in this pediatric age group. The drug is already approved for PPM as Ryjunea, licensed under Santen S.A. (搜索), in the European Union and United Kingdom.
Clinical Evidence from the Phase 3 STAR Study
The STAR study (NCT03918915), described by Sydnexis as the largest global clinical program to date in pediatric myopia, evaluated the efficacy and safety of SYD-101. Positive three-year findings were included in the company's NDA submission.
The study met its primary endpoint: the proportion of patients with confirmed myopic progression of -0.75D. Additionally, the key secondary endpoint of mean myopic annual progression rate (APR) met statistical significance at months 12, 24, and 36.
More recent data presented at the American Association for Pediatric Ophthalmology and Strabismus (AAPOS) annual meeting in April 2026 focused on the 0.01% dose. These results showed that SYD-101 significantly reduced myopia progression across all time points tested, met the primary efficacy endpoint at 36 months (p = 0.0226), and met the key secondary endpoint of mean myopic APR at months 12, 24, and 36.
The Path to an Advisory Committee
In its October 2025 complete response letter, the FDA acknowledged that the STAR trial met its primary endpoint and found no issues with the drug's safety or product quality. However, the agency concluded that the data "collectively suggest limited to no clinical benefit with questionable durability and robustness of the treatment effect." In essence, the FDA sought more compelling evidence of effectiveness than it had initially indicated to the company.
In response, Sydnexis submitted a formal dispute resolution request (FDRR), which has now led to the FDA's decision to convene an advisory committee.
Calls for Clinical Expertise on the Panel
Sydnexis CEO Perry Sternberg stated that the company has asked the FDA to include practicing pediatric ophthalmologists and optometrists on the advisory committee. "They understand the long-term challenges facing patients and families every day," Sternberg said. "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."
Robert S. Gold, MD, FAAP, a pediatric ophthalmologist and consultant for Sydnexis, emphasized the importance of including myopia treatment experts. "We're very happy that the FDA is going to convene an advisory committee meeting, hopefully, shortly," Gold said. "We trust it's going to be a non-biased committee with experience in treating myopia that will recognize how important it is to have this medication in our armamentarium to treat this increasingly prevalent disease."
The Unmet Need for an FDA-Approved Option
Currently, no FDA-approved pharmaceutical treatment exists for pediatric progressive myopia (搜索). As a result, compounded low-dose atropine is commonly prescribed off-label to slow myopia progression in children. However, compounded products do not undergo the standard FDA review process for safety, effectiveness, manufacturing consistency, and labeling, nor are they subject to post-marketing surveillance requirements. Compounding pharmacies typically operate under state boards of pharmacy, leading to varying levels of compliance with USP guidelines. Furthermore, compounded formulations are often not covered by insurance.
The need for an FDA-approved option has drawn support from national organizations. The AAPOS Myopia Task Force issued a statement welcoming the FDA's decision to further evaluate low-dose atropine for PPM, writing: "We support a rigorous, science-based review process that includes fair-minded clinicians with substantial experience currently treating children with progressive myopia using contemporary myopia-control therapies."
Earlier this year, a physician-directed public petition signed by over 1,000 U.S. eyecare providers called for the FDA to reconsider its rejection of SYD-101, emphasizing that "early intervention with SYD-101 may reduce the long-term burden of avoidable retinal disease and vision loss." The petition authors noted that "this level of support reflects broad national clinical consensus regarding the safety, efficacy, and public-health importance of making SYD-101 available to U.S. children."
The timeline for the advisory committee meeting and subsequent outcomes remains to be determined.