Beacon Therapeutics Secures RMAT Designation for Laru-Zova Gene Therapy in XLRP Treatment

Beacon Therapeutics' laruparetigene zovaparvovec (laru-zova), a gene therapy for X-linked retinitis pigmentosa (XLRP), has received the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA).

This designation is designed to expedite the development and review of regenerative therapies addressing serious or life-threatening conditions with unmet medical needs, based on preliminary clinical evidence. RMAT status allows enhanced communication with the FDA, including discussions on appropriate endpoints for accelerated approval.

Laru-Zova: A Promising Gene Therapy for XLRP

Laru-zova is an investigational gene therapy aimed at treating XLRP by expressing the full-length RPGR protein, which is crucial for addressing both rod and cone photoreceptor degeneration caused by the disease.

Leadership Commentary

Lance Baldo, MD, CEO of Beacon Therapeutics, emphasized the significance of this milestone:

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

Clinical Trials Supporting Laru-Zova’s Development

The RMAT designation is based on preliminary clinical data from Phase 2 DAWN and SKYLINE trials, which evaluate laru-zova’s efficacy, safety, and tolerability in patients with XLRP caused by RPGR gene mutations.

SKYLINE Trial

       • Ongoing, fully enrolled Phase 2 study

       • Randomized, controlled design

       • 14 male XLRP patients with RPGR mutations

       • Primary endpoint: Response proportion via microperimetry between treated and fellow eye at 12 months

DAWN Trial

       • Ongoing Phase 2, non-randomized, open-label study

       • Evaluates the fellow eye of XLRP patients previously treated with AAV vector-based gene therapy

       • Assesses two different dose levels of laru-zova for efficacy, safety, and tolerability

Additional Regulatory Designations for Laru-Zova

Beyond the RMAT designation, laru-zova has also been granted:

       • Fast Track designation (FDA, U.S.)

       • PRIME designation (European Medicines Agency, EU)

       • ILAP designation (Medicines and Healthcare Products Regulatory Agency, UK)

Conclusion

The FDA’s RMAT designation for laru-zova highlights the potential of this gene therapy to revolutionize XLRP treatment. As Beacon Therapeutics advances clinical trials and regulatory discussions, the future looks promising for patients suffering from this inherited retinal disease.