Beacon Therapeutics receives Regenerative Medicine Advanced Therapy designation for laruparetigene zovaparvovec (laru-zova)

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Beacon Therapeutics’ laruparetigene zovaparvovec (laru-zova) for the treatment of X-linked retinitis pigmentosa (XLRP) has been granted Regenerative Medicine Advanced Therapy (RMAT) by the US Food and Drug Administration (FDA).

According to a press release from the company,¹ RMAT designation was created to expedite the development and review of regenerative medicine therapies that have the potential to address unmet need in serious or life-threatening diseases, based on preliminary clinical evidence. Additionally, the designation allows for communications with the FDA throughout development including the opportunity to discuss appropriate endpoints to support accelerated approval.

Laru-zova is a gene therapy currently being investigated for the treatment of patients with XLRP. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.¹

Lance Baldo, MD, chief executive officer of Beacon Therapeutics, commented on the designation in a press release from the company.¹

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

The FDA’s RMAT designation for laru-zova recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials evaluating the efficacy, safety and tolerability of laru-zova in patients with XLRP caused by mutations in the RPGR gene.

SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. The study’s primary endpoint is the proportion of response by microperimetry between the study and fellow eye at month 12.

DAWN is an ongoing Phase 2, non-randomized, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess two different dose levels of laru-zova for efficacy, safety and tolerability in the target population.

In addition to the RMAT designation, Laru-zova also holds additional regulatory designations, including Fast Track designation from the FDA, PRIME designation from the European Medicines Agency, and ILAP designation from the Medicines and Healthcare Products Regulatory Agency in the UK.¹

References:

1. Beacon Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT). Press Release. Published January 28, 2025. Accessed January 29, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-granted-fda-regenerative-medicine-advanced-therapy-rmat/