botaretigene sparoparvovec

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botaretigene sparoparvovec

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Perioperative Steroid Use

Last Updated: 10/24/2024

SUMMARY

Botaretigene sparoparvovec is an investigational adeno-associated virus vector type 5 (AAV5) gene therapy being studied for the treatment of X-linked retinitis pigmentosa (XLRP) associated with disease causing sequence variants in the retinitis pigmentosa GTPase regulator (RPGR) gene.¹^,²
Implementation of a prophylactic triamcinolone regimen during the expansion phase of the phase 1/2 clinical trial (MGT009) resulted in a reduction in inflammation related adverse events (AEs).²

CLINICAL DATA

Phase 1/2 Clinical Trial

MGT009 was an open-label, phase 1/2 trial conducted to evaluate the safety and efficacy of single subretinal administration of botaretigene sparoparvovec as the primary and secondary endpoints, respectively, in patients with XLRP due to variants in RPGR gene.¹ Please see figure: Study Design.

Study Design²

Abbreviations: RPGR, retinitis pigmentosa GTPase regulator; R, randomized.

^aEligible adults were ≥16 years of age in the United Kingdom and ≥18 years of age in the United States; eligible children were ≥5 years of age and <16 years in the United Kingdom or <18 years in the United States. ^bWhile adults and children were eligible to participate in the dose expansion phase of the study, only adults were enrolled. ^c1:1:1 randomization. ^dOne participant assigned to the intermediate dose group of the dose expansion phase was inadvertently treated with the high dose

During the dose-escalation phase, inflammatory responses were observed in 2 out of 3 patients in the high-dose cohort, which were effectively treated with extension of steroid therapy.²

Following the implementation of a modified prophylactic corticosteroid regimen (sub-Tenon’s capsule injection of triamcinolone acetonide) in the dose expansion phase, there was a reduction in the frequency and severity of inflammation related AEs.² See figure: Participants with Ocular Inflammation-Related AEs by Severity of AE.

Participants with Ocular Inflammation-Related AEs by Severity of AE^a,b,²

Abbreviation: AE, adverse event.

^aA sub-Tenon’s capsule injection of triamcinolone was administered at the end of surgery as add-on therapy to standard steroid prophylaxis to help control ocular inflammation.

^bIncludes data throughout the treatment period including 6-months following treatment.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, Derwent Drug File (and/or other resources, including internal/external databases) was conducted on 10/21/2024.

References

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1	MeiraGTx UK II Ltd. Gene therapy for X-linked retinitis pigmentosa (XLRP) retinitis pigmentosa GTPase regulator (RPGR). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2017- [cited 2021 October 26]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03252847 NLM Identifier: NCT03252847.
2	Michaelides M, Besirli CG, Yang Y, et al. Phase 1/2 AAV5-hRKp.RPGR (Botaretigene sparoparvovec) gene therapy: safety and efficacy in RPGR-associated X-linked retinitis pigmentosa. [published online ahead of print June 11, 2024]. Am J Ophthalmol. 2024. doi:10.1016/j.ajo.2024.05.034.