Surabgene lomparvovec

Table of contents

• What is surabgene lomparvovec?
• How does surabgene lomparvovec work?
• Which conditions is it used or studied for?
• How is surabgene lomparvovec given?
• What do the clinical trials show?
• Who can take part in these trials?
• What are the trials measuring?
• What side effects are known?

What is surabgene lomparvovec?

Surabgene lomparvovec, also known by its development code ABBV-RGX-314, is an investigational gene therapy.^[3] It has no marketing authorisation in the product records, meaning it is still being studied and is not available as an approved medicine.^[3] The substance is classified as structurally diverse, of non-chemical and non-protein origin, and is supplied by AbbVie Deutschland GmbH & Co. KG.^[3] Its therapeutic area is eye diseases.^[3]

How does surabgene lomparvovec work?

Surabgene lomparvovec is a gene therapy designed to deliver genetic material into cells of the eye. The aim is to enable the eye to produce a therapeutic protein continuously, potentially reducing the need for repeated injections of anti-VEGF medicines such as ranibizumab (Lucentis) or aflibercept (Eylea).^[3][1] In nAMD, abnormal blood vessels grow under the retina and leak fluid, damaging central vision. Anti-VEGF medicines block a growth factor called vascular endothelial growth factor (VEGF) that drives this process.^[3]

Which conditions is it used or studied for?

• Neovascular age-related macular degeneration (nAMD) — a form of macular degeneration in which abnormal blood vessels grow under the retina, causing vision loss. All three trials listed study surabgene lomparvovec in this condition.^[3][2][1]

How is surabgene lomparvovec given?

Surabgene lomparvovec is formulated as a suspension for injection and is administered by subretinal use, meaning it is injected under the retina.^[3] This is a surgical procedure performed in the eye. The dose in the registry records is reported as 0 µl, which means the specific dose was not stated in the register.^[3][2][1]

What do the clinical trials show?

Three authorised clinical trials are listed for surabgene lomparvovec, all in neovascular age-related macular degeneration.^[3][2][1] The largest is a Phase 3 study (NCT05407636) with 714 participants, comparing the gene therapy against aflibercept.^[3] A long-term follow-up study (NCT03999801) includes 1,385 participants and focuses on long-term safety.^[2] A third trial (2024-512298-28-00) with 561 participants compares surabgene lomparvovec against ranibizumab, measuring injection burden and long-term visual acuity.^[1] All three trials are in the authorised stage, meaning they have received regulatory approval to proceed but are not yet completed.^[3][2][1]

Who can take part in these trials?

The trials enrol adult participants with neovascular age-related macular degeneration. The Phase 3 trial and the EU trial specify adult participants.^[3][1] The long-term follow-up study includes participants who previously received surabgene lomparvovec in earlier studies, as well as a fellow eye substudy.^[2]

What are the trials measuring?

The Phase 3 trial measures the mean change from baseline in best corrected visual acuity (BCVA) at Week 54, using the ETDRS score, to test whether surabgene lomparvovec is noninferior to aflibercept.^[3] The long-term follow-up study measures the incidence of ocular adverse events and serious adverse events.^[2] The EU trial measures the annualized intravitreal anti-VEGF injection rate from Week 6 through Week 54.^[1]

What side effects are known?

The trial records do not provide a list of side effects. The long-term follow-up study is specifically designed to monitor ocular adverse events and serious adverse events, which means safety information is being collected.^[2] No supplementary sources with detailed side effect data were provided.