FDA Grants Regenerative Medicine Advanced Therapy to Laru-Zova for XLRP

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laruparetigene zovaparvovec (laru-zova) gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP).1

Announced by Beacon Therapeutics, on January 28, 2025, the FDA’s RMAT designation expedites the development and review of therapies with the potential to address an unmet need in serious or life-threatening diseases and allows enhanced communications with the agency throughout development.

“The FDA’s decision to grant RMAT designation to laru-zova is a significant milestone for the XLRP patient community, and underscores our promising data and the potential for laru-zova to significantly improve the lives of patients who suffer from XLRP,” said Lance Baldo, MD, chief executive officer of Beacon Therapeutics.

XLRP is a severe inherited retinal disease characterized by early onset and rapid vision loss, with the potential to reach legal blindness by middle age, particularly in males. XLRP has an estimated prevalence of 1 in 25,000 males in the US, Europe, and Australia. The disease is commonly caused by mutations in the retinitis pigmentosa regulator (RPGR) gene, accounting for ≥70% of XLRP-affected families.

Laru-zova is an investigational gene therapy expressing the full-length RPGR protein and designed to address photoreceptor damage linked to XLRP, including rod and cone loss.

RMAT designation for laru-zova was based on preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials, including the DAWN trial revealing improvements in low-luminance visual acuity (LLVA), providing a potential clinically meaningful endpoint in XLRP. The company recently presented interim 3-month data from the Phase 2 DAWN trial, interim 24-month data from the Phase 2 SKYLINE trial, and demonstrated ongoing enrollment in the Phase 2/3 VISTA trial.

DAWN is an ongoing, non-randomized, open-label study investigating laru-zova in the fellow eye of males with XLRP previously treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The trial is targeting the efficacy, safety, and tolerability of two different dose levels in the XLRP population, as well as changes in visual function and functional vision, marking the first trial in this clinical development program to assess LLVA.

SKYLINE is an ongoing, fully enrolled, randomized, controlled study measuring the safety, efficacy, and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. Its primary endpoint is the proportion of response by microperimetry between the study and fellow eye at Month 12. VISTA is a currently enrolling, randomized, controlled, masked multi-center pivotal study assessing the efficacy, safety, and tolerability of laru-zova in 2 study groups, compared with an untreated control group.

Beacon also announced laru-zova has previously been granted the FDA’s Fast Track designation, PRIME designation from the European Medicines Agency (EMA), and ILP designations from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom.

“We look forward to working closely with the FDA on continued development activities to support an expedited pathway for laru-zova,” Baldo added.

References

1. Beacon Therapeutics granted FDA Regenerative Medicine Advanced Therapy (RMAT) . Beacon Therapeutics. January 28, 2025. Accessed January 31, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-granted-fda-regenerative-medicine-advanced-therapy-rmat/.
2. Martinez-Fernandez De La Camara C, Nanda A, Salvetti AP, Fischer MD, MacLaren RE. Gene therapy for the treatment of X-linked retinitis pigmentosa. Expert Opin Orphan Drugs. 2018;6(3):167-177. doi:10.1080/21678707.2018.1444476