FDA to convene Advisory Committee meeting on Sydnexis's pediatric myopia drug SYD-101

Sydnexis, Inc, stated in a news release that the US FDA has told the company it intends to convene an Advisory Committee meeting on SYD-101, the company's investigational therapy for slowing myopia progression. As of July 13, 2026, a meeting date has not been set.¹

The FDA said the committee will discuss aspects of Sydnexis's application, including findings from the Phase 3 STAR study. The company had submitted a Formal Dispute Resolution Request to the FDA's Office of Specialty Medicine after receiving a Complete Response Letter in October 2025.¹

"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, Sydnexis's chief executive officer, in the 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."

The STAR trial enrolled 847 children ages 3 to 14 in the US and Europe, with myopia ranging from -0.50 to -6.00 diopters and a mean baseline of -2.69 diopters. Participants were randomized 1:1:1 to vehicle or SYD-101 0.01%. The trial's primary endpoint — the proportion of patients with confirmed progression of -0.75 diopters — was met (p=0.0226), as was the key secondary end point of annual progression rate (p<0.001). The company said SYD-101 was well tolerated, with no unexpected atropine-related adverse events.¹

Recently, in an episode of From Paper to Clinic, Brianna Rhue, OD, FAAO, FSLS, weighed in on the study results and the CRL issued by the FDA as one of the optometrists in the study.²

“Not all atropine is created equally,” she said. “Someone else's .05(%) could be as effective as somebody else's .01(%). And when top people talk about rebound in all of these studies, you have to abruptly stop the drop. That's not how atropine works. The rebound looks real in these studies, but it's not, because what we're doing pharmacologically is binding to the scleral receptors. Just like if you don't abruptly stop a steroid, you don't abruptly … unhinge all these receptors that have been starved by the atropine.

“I believe atropine is really your gateway drug to getting a kid in another option,” Rhue continued.

There is currently no FDA-approved pharmaceutical treatment for PPM in the United States. Compounded low-dose atropine is used by some US physicians in the absence of an approved option, though compounded products do not go through FDA review for safety, effectiveness, manufacturing consistency or labeling, and access can depend on proximity to a compounding pharmacy and out-of-pocket cost. In June 2026, the American Medical Association resolved to support classifying myopia as a disease and to advocate for insurance coverage of evidence-based treatments that slow its progression in children.¹

"Low-dose atropine has become an essential tool for many pediatric ophthalmologists managing children with progressive myopia,” stated David G. Hunter, MD, PhD, president of the American Association for Pediatric Ophthalmology and Strabismus and ophthalmologist-in-chief at Boston Children's Hospital, in the release. “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,” stated Cheryl Chapman, OD, FAAO, in the release. “The call for a safe and effective solution was reflected in a Citizen Petition signed by more than 1000 eye care professionals urging the FDA to reconsider and expedite its review of SYD-101. As public health leaders, including the US 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 approved in the EU and UK, where it is licensed to Santen SA and marketed as Ryjunea.¹

According to background information cited by the company, PPM affects nearly one-third of children worldwide, with prevalence projected to exceed 740 million cases by 2050 (Liang et al, 2024). A 2016 study in Ophthalmology (Holden et al) projected North American myopia prevalence could reach nearly 60% by 2050. Research by Hu et al (2020) found the most rapid disease progression occurs in children ages 3 to 10, with associated risks including cataracts, glaucoma, retinal detachment and myopic maculopathy in more severe cases.¹

References:

1. Sydnexis announces FDA Advisory Committee meeting to review NDA for SYD-101 for pediatric progressive myopia. News release. July 13, 2026. Accessed July 13, 2026. https://www.businesswire.com/news/home/20260713332414/en/Sydnexis-Announces-FDA-Advisory-Committee-Meeting-to-Review-NDA-for-SYD-101-for-Pediatric-Progressive-Myopia

2. Shah H, Rhue B, Hoffman M. From Paper to Clinic, Episode 5: Reframing Myopia Management: Atropine, Axial Length, and Engaging Young Patients. Optometry Times. July 6, 2026. https://www.optometrytimes.com/view/from-paper-clinic-ep-5-myopia-management-atropine-axial-length-pediatrics