Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
Publication/Presentation Date
5-1-2023
Abstract
PURPOSE: To evaluate the safety profile of lenadogene nolparvovec (Lumevoq) in patients with Leber hereditary optic neuropathy.
DESIGN: Pooled analysis of safety data from 5 clinical studies.
METHODS: A total of 189 patients received single unilateral or bilateral intravitreal injections of a recombinant adeno-associated virus 2 (rAAV2/2) vector encoding the human wild-type ND4 gene. Adverse events (AEs) were collected throughout the studies, up to 5 years. Intraocular inflammation and increased intraocular pressure (IOP) were ocular AEs of special interest. Other assessments included ocular examinations, vector bio-dissemination, and systemic immune responses against rAAV2/2.
RESULTS: Almost all patients (95.2%) received 9 × 10
CONCLUSIONS: Lenadogene nolparvovec had a good overall safety profile with excellent systemic tolerability, consistent with limited bio-dissemination. The product was well tolerated, with mostly mild ocular side effects responsive to conventional ophthalmologic treatments.
Volume
249
First Page
108
Last Page
125
ISSN
1879-1891
Published In/Presented At
Vignal-Clermont, C., Yu-Wai-Man, P., Newman, N. J., Carelli, V., Moster, M. L., Biousse, V., Subramanian, P. S., Wang, A. G., Donahue, S. P., Leroy, B. P., Sadun, A. A., Klopstock, T., Sergott, R. C., Rebolleda Fernandez, G., Chwalisz, B. K., Banik, R., Taiel, M., Roux, M., Sahel, J. A., & LHON STUDY GROUP (2023). Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy. American journal of ophthalmology, 249, 108–125. https://doi.org/10.1016/j.ajo.2022.11.026
Disciplines
Business Administration, Management, and Operations | Health and Medical Administration | Management Sciences and Quantitative Methods
PubMedID
36496192
Department(s)
Administration and Leadership
Document Type
Article