Adverum Biotechnologies Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for Ixo-vec for the Treatment of Wet AMD
August 01 2024 - 8:00AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage
company pioneering the use of gene therapy as a new standard of
care for highly prevalent ocular diseases, today 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, M.D.,
president and chief executive officer 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 Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is a VEGF-driven
advanced form of age-related macular degeneration (AMD) associated
with the build-up of fluid in the macula and the retina. Wet AMD is
a leading cause of blindness in people over 65 years of age, with
approximately 20 million individuals worldwide living with this
condition. New cases of wet AMD are expected to grow significantly
worldwide as populations age. AMD is expected to impact 288 million
people worldwide by 2040, with wet AMD accounting for approximately
10% of those cases. Additionally, wet AMD is a bilateral disease,
and incidence of nAMD in the second eye is up to 42% in the first
two to three years. The current standard of care requires frequent
life-long repeated bolus injections of anti-VEGF in the eye.
Intravitreal (IVT) gene therapy has the promise to preserve vision
and reduce most or all injections for the life of the patient by
delivering stable therapeutic levels of anti-VEGF to control fluid
associated with the disease.
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.
About Adverum Biotechnologies
Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage
company that aims to establish gene therapy as a new standard of
care for highly prevalent ocular diseases with the aspiration of
developing functional cures to restore vision and prevent
blindness. Leveraging the capabilities of its proprietary
intravitreal (IVT) platform, Adverum is developing durable,
single-administration therapies, designed to be delivered in
physicians’ offices, to eliminate the need for frequent ocular
injections to treat these diseases. Adverum is evaluating its novel
gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly
referred to as ADVM-022), as a one-time, IVT injection for patients
with neovascular or wet age-related macular degeneration.
Additionally, by overcoming the challenges associated with current
treatment paradigms for debilitating ocular diseases, Adverum
aspires to transform the standard of care, preserve vision, and
create a profound societal impact around the globe. For more
information, please visit www.adverum.com.
Inquiries:
Adverum Investor Relations
Email: ir@adverum.com
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