Muscular Dystrophy Association Applauds Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above
June 20 2024 - 6:42PM
The Muscular Dystrophy Association (MDA) celebrates the U.S. Food
and Drug Administration’s expanded approval of ELEVIDYS
(delandistrogene moxeparvovec-rokl). The efficacy supplement
approval will expand the labeled indication of ELEVIDYS to include
boys and men with Duchenne muscular dystrophy (DMD) with a
confirmed mutation in the DMD gene who are at least 4 years of age.
The FDA granted traditional approval for ambulatory patients and
accelerated approval for non-ambulatory patients, confirming the
functional benefits of the treatment.
Developed by Sarepta Therapeutics, ELEVIDYS targets the genetic
root cause of Duchenne muscular dystrophy (DMD) and works by
introducing an engineered gene that codes for a shortened version
of dystrophin, referred to as ELEVIDYS micro-dystrophin into muscle
cells. The FDA granted accelerated approval for boys ages 4 and 5
in June of 2023. Read more in the Sarepta press release.
“The FDA’s expanded approval for ELEVIDYS marks a
pivotal moment for the community, offering renewed hope and
tangible progress in our fight against DMD,” said Sharon
Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy
Association. "At the Muscular Dystrophy Association, we
continue our legacy and impact with innovative scientific and
clinical research to propel treatments and therapies forward for
the families we serve.”
"Today's FDA approval of ELEVIDYS is a significant step forward
in the fight against Duchenne muscular dystrophy. The accelerated
approval expansion is not only a scientific accomplishment but a
major source of hope for countless families affected by this
relentless disease. As the Chief Medical Advisor of Muscular
Dystrophy Association, I am proud to stand with our community in
celebrating this milestone. Together, we are transforming what is
possible in medical research and patient care, reaffirming our
commitment to improving lives and finding a cure," said Barry
Byrne, MD, PhD, Chief Medical Advisor, Muscular Dystrophy
Association, and Associate Chair of Pediatrics and Director of
the Powell Gene Therapy Center at the University of Florida.
“By expanding the label beyond current age groups, the FDA is
exercising flexibility in its approach to a rare disease with high
unmet need. Many older boys and young men urgently need access to
new therapies that could alter the disease's progression. By
broadening the indication, hope can be offered to more families and
improve the quality of life for a larger segment of the DMD
community”, said Paul Melmeyer, EVP, Public Policy and Advocacy,
MDA. "Furthermore, transitioning from accelerated approval to
full approval for the ambulatory population is impactful as it
solidifies the evidence of ELEVIDYS' clinical benefits and
facilitates continued access to this critical therapy. Confirming
the therapy’s effectiveness will provide greater assurance to
families and healthcare providers, enhancing the long-term
treatment landscape for Duchenne muscular dystrophy (DMD). While
the expanded approval of ELEVIDYS marks a significant milestone, we
must acknowledge the individuals who will not be included within
the current label. Our commitment remains steadfast to continue
advocating for inclusive research and development efforts that
strive to expand access to all individuals living with DMD,
ensuring no one is left behind."
“There weren’t many options when Conner was diagnosed with
Duchenne muscular dystrophy nine years ago but with the MDA they’re
accelerating research, and he did get gene therapy. I believe he
would be in a wheelchair now full time if he hadn’t received it.
Muscular Dystrophy Association was an intricate part of that
process,” said Jessica Curran, MDA family member.
MDA remains steadfast in its mission to accelerate the
development of treatments and cures for neuromuscular diseases. The
organization recently announced expanded support for the families
and clinical community through the, which provides guidance and
support on novel gene therapy treatments, including ELEVIDYS.
MDA implemented the MDA Gene Therapy Support Network (GTxSN) to
support the MDA Care Center Network and the neuromuscular (NMD)
community address the challenges and achieve the promise of gene
therapy. An element of the GTxSN is to establish a clinical
operational readiness network to support existing and new gene
therapy treatments for the NMD patient community within the MDA
Care Center Network. To help evaluate and expand gene therapy
access in a way that optimizes safety and long-term outcomes, MDA
created a dedicated Care Center Network Information Hub (CCNH) to
host educational resources and clinical best practices sharing
amongst healthcare providers within the MDA Care Center Network.
The CCNH fosters greater cross-clinic engagement and gene therapy
best practice sharing; focus on real time expert learnings and
experiences (i.e. best practices, protocols, infrastructure check
lists, etc.).
With the help of the MDA GTxSN, the goal is for MDA Care Center
clinicians and staff to feel that they are ready and confident to
dose patients and perform post-administration monitoring of
patients, minimize the number of adverse events experienced post
gene therapy at MDA Care Center sites, effectively manage and
schedule resources at MDA Care Centers sites to maximize the number
of patients treated per month, and celebrate success stories from
patients and families post-treatment.
MDA has been at the forefront of advancements in DMD research
for over 70 years, investing nearly $227 million in DMD research
and clinical trials. Recent collaborations with CureDuchenne and
Parent Project Muscular Dystrophy have focused on strategizing
approaches to reduce anti-AAV antibodies to enable safe and
effective delivery of gene therapy in DMD patients. Historic
contributions include discovering the DMD-causing gene in the
1980s. Recognized by The American Society for Gene and Cell
Therapy, MDA has invested over $125 million in gene therapy
development. The organization’s dedication earned them the Sonia
Skarlatos Public Service Award in 2019 for their impactful
contributions to genetic medicine.
About Muscular Dystrophy AssociationMuscular Dystrophy
Association (MDA) is the #1 voluntary health organization in
the United States for people living with muscular dystrophy, ALS,
and related neuromuscular diseases. For over 70 years, MDA has led
the way in accelerating research, advancing care, and advocating
for the support of our families. MDA's mission is to empower the
people we serve to live longer, more independent lives. To learn
more visit mda.org and follow MDA on Instagram,
Facebook, X, Threads, TikTok, LinkedIn,
and YouTube.
- FDA Approval of ELEVIDYS for Duchenne Muscular Dystrophy
Patients Ages 4 and Above
Mary Fiance, Vice President, Strategic Communications
Muscular Dystrophy Association
press@mdausa.org