Sydnexis has revealed further data of its paediatric progressive myopia (PPM) candidate, which met both the primary and secondary endpoints, just days after it was turned down by the US Food and Drug Administration (FDA).
The agency issued Sydnexis with a complete response letter (CRL) for SYD-101 in PPM on 23 October. While the FDA acknowledged that the study met this primary endpoint, the letter stated that the data do not support the effectiveness of low-dose atropine in children with myopia.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The application was made on the basis of results from the Phase III STAR trial (NCT03918915), for which Sydnexis revealed more data during a poster session at the Academy of Managed Care Pharmacy (AMCP) Nexus 2025 in National Harbor, Maryland.
Data presented at AMCP showed that the magnitude of clinical effect of change in spherical equivalent (SE) was greatest in younger patients, aged three to 12 years at treatment initiation, with both doses demonstrating nominal statistical significance.
In the full cohort, SE change after treatment with SYD-101 0.01% and 0.03% was -0.71 and 0.76, respectively, compared to ‑0.92 in the vehicle cohort.
Also presented at AMCP was data in a subgroup of fast progressors. In these patients with low to moderate myopia, SYD-101 0.01% reduced the progression by more than 50%.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataSydnexis CEO Perry Sternberg said: “The data presented at AMCP Nexus 2025 show strong treatment effects in the full study population at 36 months, but in younger patients, the magnitude of effect gets larger, and in patients with documented faster progression, the magnitude of treatment effect increases even more.”
The company previously announced that the candidate met the primary endpoint of proportion of patients with confirmed progression and a key secondary endpoint of mean myopic annual progression rate. These endpoints were both met despite low dose patients aged 16 to 18 exhibiting very little progression.
Despite the FDA refusal, SYD-101 did receive approval in the European Union (EU) in June 2025, where it is marketed as Ryjunea as part of a partnership with Santen Pharmaceutical. Santen also holds the rights to commercialisation in the Middle East and Africa.
More commonly known as short-sightedness, myopia is commonly managed with contact lenses or glasses. In paediatric patients, low-dose atropine drops may be used in an attempt to slow progression, as well as orthokeratology (ortho-k) contact lenses, which are worn overnight to flatten to front surface of the eye.
In 2024, Eyenovia’s stock crashed after its drug-device combination for myopia failed to meet the primary endpoint of a Phase III trial.
