Adverum Biotechnologies, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Ixo-vec, its clinical-stage gene therapy product candidate, for the treatment of wet AMD.
“The RMAT designation is based on the clinical data from Ixo-vec to date and recognizes the transformative potential of our gene therapy for patients with wet AMD,” stated Laurent Fischer, MD, president and CEO of Adverum Biotechnologies. “This validates that Ixo-vec has the potential to address the significant unmet medical needs in treating nAMD. With both RMAT and PRIME designations, we look forward to continuing our close collaborations with both the FDA and EMA on our pivotal program and to sharing updates from regulatory discussions in the fourth quarter of this year.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies.
A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.
The RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review.
About Ixo-vec in wet AMD
Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette.
Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician’s office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF injections into the eye, optimize patient compliance and improve vision outcomes for patients with wet AMD.
Ixo-vec is currently being evaluated in the ongoing phase 2 clinical trial LUNA (NCT05536973) and the phase 1 OPTIC extension study (NCT04645212) for the treatment of neovascular or wet AMD.
In recognition of the need for new treatment options for wet AMD, FDA granted Fast Track and RMAT designations for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the EMA and the Innovation Passport from the United Kingdom’s Medicines and Healthcare products Regulatory Agency for the treatment of wet AMD.