The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ophthalmic gene therapy company Beacon Therapeutics’ laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP).
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. RMAT designation provides benefits including interactive communications with the FDA throughout development, the opportunity to discuss appropriate endpoints to support accelerated approval, and eligibility for expedited programs such as priority review.
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. This evidence includes emerging data from DAWN showing improvements in low luminance visual acuity (LLVA), which provides support for the measure as a clinically meaningful endpoint in XLRP.
“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. “We look forward to working closely with the FDA on continued development activities to support an expedited pathway for laru-zova.”
Laru-zova also holds several 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.
Beacon Therapeutics recently presented updates on several studies of laru-zova, including positive interim 3-month data in the Phase 2 DAWN trial, positive interim 24-month data from the Phase 2 SKYLINE trial, and ongoing enrollment in the Phase 2/3 VISTA trial.
