FDA accepts new drug application for pharmaceutical aiming to treat pediatric myopia

Sydnexis announced that the FDA accepted its new drug application for SYD-101, a pharmaceutical intended to treat progressive pediatric myopia. The FDA set a Prescription Drug User Fee Act (PDUFA) target action date for October 23. If approved, according to the company, this would become the first and only currently available pharmaceutical for treatment of progressive pediatric myopia in the U.S. The submission is based on the company’s Phase 3 study, which enrolled more than 850 patients aged 3 to 14 years old. The company called it the “largest clinical study ever completed for the treatment of progression of pediatric myopia.” The company described SYD-101 as a “proprietary low-dose atropine formulation.”

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FDA approves treatment for macular telangiectasia type 2

Neurotech Pharmaceuticals announced FDA approval of ENCELTO (revakinagene taroretcel-lwey), a gene therapy implant, for the treatment of macular telangiectasia type 2 (MacTel). This is, according to the company, the first and only treatment for this indication. In Phase 3 clinical trials, ENCELTO was shown to significantly slow the loss of macular photoreceptors over 24 months. It is expected to be available in the U.S. beginning in June 2025.

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Survey reveals perspectives of patients with glaucoma

The Preservative Freedom Coalition announced the results of a survey of 558 patients with glaucoma or ocular hypertension. Among them were that: 1 in 5 respondents were satisfied with their current treatment; about 33% stopped, skipped, or changed medication due to “undesirable impacts or negative effects”; 60% were concerned about toxicity of preservatives; 61% were unaware of preservative-free treatment alternatives; and 62% reported symptoms like gritty eyes and discomfort. The Preservative Freedom Coalition reported in its press release that the results “underscore the critical need for improved dialogue between glaucoma patients and their eye care providers, specifically around preservatives and their eye health.”

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New wearable vision testing platform

Topcon Healthcare, RadiusXR, and Glaukos Corporation announced their collaboration and launch of RadiusXR’s new vision testing platform—Inspire. This platform, according to the company’s press release, “unites RadiusXR’s innovative visual field platform and Topcon Healthcare’s global leadership in robotic diagnostics and digital healthcare solutions with Glaukos’ commitment to expanding patient access by democratizing the diagnosis of ophthalmic diseases such as glaucoma.” The Inspire platform integrates “wearable vision testing with comprehensive ophthalmic imaging and AI-driven data analysis.”

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Seeking Fast Track Designation for investigational treatment of neuropathic corneal pain

OKYO Pharma announced that it filed an application with the FDA for a Fast Track Designation for urcosimod for treatment of neuropathic corneal pain. According to the company’s press release, urcosimod (OK-101) is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor found on the immune cells in the eye associated with inflammatory response, in addition to on neurons and glial cells in the dorsal root ganglion. It has been shown to produce anti-inflammatory and pain-reducing benefits in mouse models of dry eye disease and corneal neuropathic pain, respectively. It is currently under Phase 2 investigation for neuropathic corneal pain among 48 patients who were randomized to receive the treatment or placebo. Fast Track Designation, if granted, would speed the development and review of urcosimod.

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Fast Track Designation granted to gene therapy targeting X-linked retinoschisis

Atsena Therapeutics announced that it received an FDA Fast Track Designation for its gene therapy ATSN-201 for treatment of X-linked retinoschisis (XLRS).

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ASCRS news and events

• 2025 ASCRS Annual Meeting: Registration is open for the ASCRS Annual Meeting, taking place April 25–28, in Los Angeles, California. Find the most up-to-date information on the Real-Time Program here.
• SightLine at ASCRS: This is a new day-long meeting April 24, ahead of the ASCRS Annual Meeting, for all commercial stakeholders in ophthalmology to learn about and collaborate on technologies and care delivery ideas that will forge a more sustainable financial future. Learn more.
• Business of Interventional Glaucoma: Surgeons and practice administrators alike are invited to the ASOA ASCRS Business of Interventional Glaucoma program, taking place Thursday, April 24 from 1:00–5:30 p.m. in Los Angeles ahead of the ASCRS Annual Meeting and ASOA Annual Meeting. Learn more.
• ASCRS 50th anniversary: ASCRS members from the 50 states are sending in their perspectives on the Society, its impact on their career, and its influence on the specialty as a whole. Tune in each week through April 2025 for a new video.

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Research highlights

• A study published in Nature Communications described a Phase 1/2, single-center, single-arm study evaluating use of cultivated autologous limbal epithelial cells (CALEC) for treatment of blindness caused by limbal stem cell deficiency. According to the paper’s abstract, “CALEC grafts met release criteria [a feasibility outcome] in 14 (93%) of 15 participants at conclusion of trial.” There was one bacterial infection, which the authors reported was unrelated to treatment; no other primary safety events occurred. The authors reported that 86%, 93%, and 92% of grafts achieved complete or partial success at 3, 12, and 18 months, respectively. The authors report that these results “support that CALEC transplantation is safe and feasible,” but they noted further research is needed to evaluate therapeutic efficacy.
• A retrospective, consecutive case series determined the visual and refractive outcomes of eyes with a history of radial keratotomy that later received a second-generation Light Adjustable Lens (LAL, RxSight). Ninety-four eyes from 77 patients were included in the study, 26 of which (28%) had a history of 4-cut RK, 11 (12%) had 6-cut RK, 52 (55%) had 8-cut RK, and 5 (5%) had 16-cut RK. Eighty-two percent of eyes achieved 20/25 UDVA or better, and 74% achieved a UDVA of 20/20 or better. Ninety-four percent were correctable to 20/20 or better. Ninety-eight percent of eyes were within 1 D of target, 88% were within 0.5 D of target, and 69% within 0.25 D of target. The authors concluded that patients with prior RK achieved “favorable visual and refractive outcomes” with the LAL. They advised postop light adjustments be delayed, allowing for refractive stabilization. The study is published in the Journal of Cataract & Refractive Surgery.

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Product news

• Harrow announced that it has begun developing its next-generation of TRIESENCE (triamcinolone acetonide injectable suspension). It plans to submit a new drug application to the FDA in 2027.
• Optos launched a reference database for its Monaco and MonacoPro combined ultra-widefield and spectral domain OCT devices. This is, according to the company, the largest reference database for ultra-widefield and OCT imaging.

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