Ophthalmic gene therapy company Beacon Therapeutics Holdings Limited announced on Tuesday that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP).

The FDA's RMAT designation for laru-zova recognises 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.

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.