Nimesh Patel, MD, on low-dose atropine and SYD-101

Nimesh A. Patel, MD, said he is hopeful the FDA will approve SYD-101 (atropine 0.01%; Sydnexis) for pediatric progressive myopia because its phase 3 trial met its end points. The FDA will review the application at an advisory committee meeting, including an open public hearing, on October 30, 2026.¹ Patel, director of pediatric retina at Mass Eye and Ear and Mass General Hospital and director of pediatric retina surgery at Boston Children's Hospital, discussed compounded atropine and the regulatory path for SYD-101 with the Eye Care Network.

Why does myopia control matter beyond refractive correction?

Patel said myopia prevention has drawn growing attention because complications extend into adulthood, including myopic degeneration, choroidal neovascularization, and retinal detachment. He also noted that high myopia limits daily function without glasses or contact lenses, and that contact lens wear carries a risk of corneal ulcers. Among prevention strategies, which include orthokeratology, he identified low-dose atropine as probably the most popular and one of the most studied.

What are the risks of compounded low-dose atropine?

Because no FDA-approved low-dose atropine formulation exists in the US, clinicians rely on compounded preparations. Patel said compounded atropine works but raised concerns about cost, insurance coverage, and standardization, citing studies that found some compounded formulations contain no more than 80% to 90% of the intended drug. The compounded drug is "still relatively safe and probably effective, it's just not as well regulated, not available through insurance and potentially costly," he told the Eye Care Network.

Where does SYD-101 stand with the FDA?

The FDA issued a complete response letter for SYD-101 in October 2025, acknowledging that the phase 3 STAR trial (NCT03918915) met its primary end point but concluding that the data did not support effectiveness.² STAR enrolled 847 children aged 3 to 14 years.¹ SYD-101 is approved in the European Union and United Kingdom, where Santen markets it as Ryjunea.¹

What makes a benefit clinically meaningful?

Patel said the trials were designed in discussion with regulators and met their end points, which he considers generally sufficient for approval. "It's hard to get there in a clinical trial setting, to be honest," he said, citing enrollment across varied levels of progression, limited study duration, and participants who reached ages 16 to 18 years by study end, when he said myopia is unlikely to progress. He said the benefit was quite clinically significant among fast progressors and younger children. "But if it's being used already in an unregulated compounded form, I think the assumption is that it's probably already clinically beneficial," Patel said.

What message does Patel have for regulators and clinicians?

Patel said the most underestimated aspect of pediatric progressive myopia is its link to long-term complications. "Myopia is associated with all these other long-term, real vision-threatening things that glasses and contacts cannot fix," he said. He supports the FDA's role in ensuring treatments are safe and effective, but once that standard is met, he said, "we want it in the hands of the doctors."

References
1. Sydnexis. Sydnexis announces FDA advisory committee meeting date to review NDA for SYD-101 for pediatric progressive myopia. News release. September 24, 2026. Accessed September 25, 2026. https://www.biospace.com/press-releases/sydnexis-announces-fda-advisory-committee-meeting-date-to-review-nda-for-syd-101-for-pediatric-progressive-myopia
2. Sydnexis. Sydnexis receives complete response letter from FDA for SYD-101 to slow pediatric myopia progression. News release. October 23, 2025. Accessed September 25, 2026. https://sydnexis.com/sydnexis-receives-complete-response-letter-from-fda-for-syd-101-to-slow-pediatric-myopia-progression/