Company remains committed to ongoing public dialogue on the clinical evidence supporting SYD-101 and the real-world challenges facing children, families, and eye care professionals
DEL MAR, Calif.--(BUSINESS WIRE)--Sydnexis, Inc., a biopharmaceutical company focused on developing a novel low-dose atropine formulation to treat pediatric progressive myopia (PPM), today announced that the U.S. Food and Drug Administration (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 PPM. 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 the Company’s application, including findings from the Phase 3 STAR study. Sydnexis submitted a Formal Dispute Resolution Request (FDRR) to the FDA’s Office of Specialty Medicine (OSM) after receiving a Complete Response Letter (CRL) in October of 2025.
The Phase 3 STAR trial is the largest global clinical program completed to date in pediatric myopia. It evaluated a broad population of 847 children aged 3–14 at treatment initiation. Participants with myopia of -0.50 diopters (D) to -6.00 D, with a mean baseline of -2.69 D, were enrolled across the U.S. and Europe and randomized (1:1:1) to vehicle (placebo) and SYD-101 0.01%. The study’s 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 the key secondary endpoint (p<0.001). Additionally, SYD-101 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. "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."
Compounded low-dose atropine, supported by emerging evidence of clinically proven efficacy, is currently used by many U.S. physicians to manage PPM in the absence of an FDA-approved pharmaceutical treatment option. While compounded atropine has filled a critical gap in care, compounded products do not undergo the FDA review process for safety, effectiveness, manufacturing consistency, and labeling, and do not have the same post-marketing surveillance requirements as an approved prescription medicine. Access to compounded therapies can also vary based on factors such as proximity to a compounding pharmacy and the ability to pay for a prescription out-of-pocket, creating additional barriers for children in need of treatment. In June 2026, the American Medical Association formally resolved to support the classification of myopia as a disease and to advocate for comprehensive insurance coverage of evidence-based treatments that slow its progression in children.
"Low-dose atropine has become an essential tool for many pediatric ophthalmologists managing children with progressive myopia," said David G. Hunter, MD, PhD, President of the American Association for Pediatric Ophthalmology and Strabismus (AAPOS) and Ophthalmologist-in-Chief at Boston Children's Hospital. "For younger children at risk of developing high myopia, treatment options remain extremely limited to help slow progression. Low-dose atropine has generated significant use within the clinical community because its therapeutic advantage has been seen over years of real-world use. However, physicians, parents, and patients would benefit greatly from access to an FDA-approved option supported by consistent manufacturing standards, labeling, and heightened regulatory oversight."
"Progressive myopia is rapidly becoming the most common pediatric eye disease, as more children develop the condition at younger ages and face a greater risk of serious and permanent vision complications," said Cheryl Chapman, OD, FAAO. "The call for a safe and effective solution was reflected in a Citizen Petition signed by more than 1,000 eye care professionals urging the FDA to reconsider and expedite its review of SYD-101. As public health leaders, including the U.S. Surgeon General, continue to raise concerns about the impact of screen use and near work on children’s vision, the need for an FDA-approved treatment option has only become more important."
SYD-101 is currently approved in the European Union and UK, where it is licensed to Santen S.A. and marketed as Ryjunea®.
About Pediatric Progressive Myopia (PPM)
Pediatric Progressive Myopia (PPM) is the most common eye disease in children and a rapidly rising global health concern. Nearly one-third of children worldwide are already affected by this degenerative disease, with prevalence projected to exceed 740 million cases by 2050 (Liang et al., 2024). In North America, myopia prevalence is expected to reach nearly 60% by 2050, according to a landmark study published in Ophthalmology (Holden et al., 2016). The most rapid progression of the disease occurs in children ages 3 to 10 years (Hu et al., 2020), with patients who start progressing younger experiencing more severe outcomes and associated co-morbidities, including cataracts, glaucoma, retinal detachment, and myopic maculopathy. Despite its growing prevalence, there are currently no FDA-approved pharmaceutical options in the United States to slow the progression of PPM, representing a significant gap in available treatment options for patients.
About Sydnexis, Inc.
Sydnexis, Inc. is a biopharmaceutical company dedicated to advancing care for pediatric progressive myopia. The company’s lead compound, SYD-101, is currently approved in the European Union and UK, where it is licensed to Santen S.A. and marketed as Ryjunea®. Sydnexis is supported by leading life-science investors, including Visionary Ventures, RA Capital, Longitude Capital, and Bluestem Capital. For more information on Sydnexis, please visit www.sydnexis.com.
Contacts
FTI Consulting
Robert Stanislaro or Helen O’Gorman
sydnexis@fticonsulting.com
