What the label lists and what reports show
Voretigene neparvovec-rzyl side effects
Voretigene neparvovec-rzyl's FDA label: “The most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and …” FAERS holds 152 reports naming it through July 30, 2026; the reaction reported most often, leaving out the conditions it is used for, product-use terms and outcomes, is intraocular pressure increased (22 reports).
- No generic listed
Check it at the source: the voretigene neparvovec-rzyl FDA label on DailyMed (effective May 1, 2022); the FDA FAERS public dashboard.
What does the label list?
From the label: adverse reactionsThe most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula).
Table 1. Ocular Adverse Reactions Following Treatment with LUXTURNA (N=41)
| Adverse Reactions | Subjects n=41 | Treated Eyes n=81 |
|---|---|---|
| Any ocular adverse reaction | 27 (66%) | 46 (57%) |
| Conjunctival hyperemia | 9 (22%) | 9 (11%) |
| Cataract | 8 (20%) | 15 (19%) |
| Increased intraocular pressure | 6 (15%) | 8 (10%) |
| Retinal tear | 4 (10%) | 4 (5%) |
| Dellen (thinning of the corneal stroma) | 3 (7%) | 3 (4%) |
| Macular hole | 3 (7%) | 3 (4%) |
| Subretinal deposits | 3 (7%) | 3 (4%) |
| Eye inflammation | 2 (5%) | 4 (5%) |
| Eye irritation | 2 (5%) | 2 (2%) |
| Eye pain | 2 (5%) | 2 (2%) |
| Maculopathy (wrinkling on the surface of the macula) | 2 (5%) | 3 (4%) |
| Foveal thinning and loss of foveal function | 1 (2%) | 2 (2%) |
| Endophthalmitis | 1 (2%) | 1 (1%) |
| Foveal dehiscence (separation of the retinal layers in the center of the macula) | 1 (2%) | 1 (1%) |
| Retinal hemorrhage | 1 (2%) | 1 (1%) |
Rows and numbers as the label prints them, under its own column headings (the first rows of a longer table where it runs on). A reaction in a trial table is not necessarily caused by the medicine, and trials differ from everyday use.
From the label: adverse reactions, continuedThe most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). The most common adverse reactions (incidence ≥ 5%) in the clinical trials were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Spark Therapeutics, Inc. at 1-855-SPARKTX, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of other products and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to LUXTURNA in two clinical trials consisting of 41 subjects (81 eyes) with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Forty of the 41 subjects received sequential subretinal injections of LUXTURNA to each eye. …
Source: FDA drug label, Spark Therapeutics, Inc., effective May 1, 2022 (SPL set 08313a24-e7ce-457a-bb3f-161bc45517ee), via openFDA; the full label on DailyMed
What do FAERS reports show?
152 reports list voretigene neparvovec-rzyl among the medicines taken, each a report of something that happened, not proof the medicine caused it; 1 are coded as a death and 15 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is. Of the 24 reactions below, 9 are named by the same term (in US spelling) in the label's adverse reactions, warnings or patient leaflet; a reaction the label does not name may still be there under another word, and a report does not show that the medicine caused it.
The 25 most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026; reactions as MedDRA preferred terms
What next?
- Voretigene neparvovec-rzyl: the full record
- label, generics, price, shortages, recalls
- Report a side effect to the FDA
- MedWatch
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