WASHINGTON, June 20,
2024 /PRNewswire/ -- Parent Project Muscular
Dystrophy (PPMD), a nonprofit organization leading the fight to end
Duchenne muscular dystrophy (Duchenne), applauds the U.S. Food and
Drug Administration (FDA) for its decision to expand the labeled
indication for ELEVIDYS, a micro-dystrophin gene therapy, to
include individuals at least 4 years of age and remove ambulation
restrictions, marking a significant milestone in the Duchenne
treatment landscape. Additionally, the FDA has converted the
ELEVIDYS accelerated approval to a traditional approval for
ambulatory patients. The indication in non-ambulatory patients is
under the accelerated approval pathway and will require
confirmatory studies to convert to traditional approval.
The ELEVIDYS label now states that the adeno-associated virus
(AAC) vector-based gene therapy is indicated in individuals at
least 4 years of age: "For the treatment of Duchenne muscular
dystrophy (DMD) in patients who are ambulatory and have a confirmed
mutation in the DMD gene," as well as, "For the treatment of
DMD in patients who are non-ambulatory and have a confirmed
mutation in the DMD gene," expanding the label previously
approved and filed with the FDA through Accelerated Approval in
June 2023. This expanded indication
offers hope and opportunity for a broader spectrum of individuals
living with Duchenne and their families.
"This decision by the FDA represents a monumental leap forward
in our collective efforts to end Duchenne muscular dystrophy," said
PPMD Founding President and CEO Pat
Furlong. "By expanding the label's age inclusion and
removing ambulation restrictions, ELEVIDYS can now reach and
benefit a more diverse population of Duchenne patients, providing
more families with the opportunity to consider this treatment as an
option for their child or themselves."
ELEVIDYS is made available and marketed in the United States by Sarepta Therapeutics. The
FDA had previously granted an Accelerated Approval to ELEVIDYS for
patients ages 4-5 years old in June
2023. In February 2024, FDA
accepted and filed Sarepta Therapeutics' efficacy supplement to the
Biologics License Application (BLA) for ELEVIDYS, with the efficacy
supplement seeking to expand the therapy's labeled indication.
During its review, FDA also evaluated the efficacy supplement to
convert the ELEVIDYS accelerated approval to a traditional
approval. FDA approval of a drug means that data on the drug's
effects have been reviewed by the Center for Drug Evaluation and
Research (CDER), and the drug is determined to provide benefits
that outweigh its known and potential risks for the intended
population. After review of the efficacy supplement, FDA has
converted the ELEVIDYS accelerated approval to a traditional
approval for ambulatory patients. ELEVIDYS is approved for
non-ambulatory patients through the Accelerated Approval pathway
based on the surrogate outcome of micro-dystrophin expression.
Continued approval for this indication may be contingent upon
verification and description of clinical benefit in a confirmatory
trial.
"We extend our sincere appreciation to the FDA for its
flexibility and dedication to patient-centric decision-making and
its commitment to advancing therapies for rare diseases.
Furthermore, we are eternally grateful to the Duchenne
community, including patients, families, clinicians, researchers,
and advocates, whose unwavering support and collaboration have been
instrumental in driving progress in the fight to end Duchenne,"
Furlong added. "Patients and families need treatment options, and
we applaud the expansion of these options. We also recognize this
is just the beginning for AAV-based gene therapies, and we eagerly
look forward to continued innovation and progress."
PPMD has long been optimistic that gene therapy could be a
potential treatment for Duchenne. PPMD launched its Gene Therapy
Initiative in 2017 as a long-term concept seeking to accelerate the
potential of gene therapy as a therapeutic for Duchenne. Its early
strategy was to bring attention to and fund key questions that must
be answered in order for the technology to progress toward
approvals. Since the launch of the Initiative, PPMD has funded over
$7 million in a variety of gene
therapy and related approaches to several institutions, including
the work of Dr. Jerry Mendell at
Nationwide Children's Hospital's Abigail Wexner Research Institute,
which led to the development of ELEVIDYS.
The label for ELEVIDYS currently excludes patients who are
younger than age 4. Sarepta has ongoing studies in these patients
to determine if ELEVIDYS is safe and effective. Additionally,
ELEVIDYS is currently not available for people with DMD gene
deletions in exons 8 and/or 9 due to safety concerns.
Sarepta will participate in PPMD's 30th Annual Conference in
Orlando, Florida, taking place
June 27-29, 2024, where we look
forward to learning more about the expanded label.
For three decades, PPMD has been dedicated to working with
researchers, clinicians, industry, and families to advance
treatments for all people living with Duchenne and Becker. The
organization employs a comprehensive approach to identify
opportunities to accelerate development of all therapies for
Duchenne, including regulatory, research, and patient-recruitment
counsel to help expedite the progress of investigational
products.
PPMD's patient-report registry, The Duchenne Registry, as well
as PPMD's Duchenne Outcomes Research Interchange, serve as
resources for researchers and industry to access aggregated,
de-identified data provided by patients and families. Additionally,
PPMD remains committed to its innovative patient engagement
efforts, which include multiple patient preference studies and a
revised community-generated draft guidance document, recently
updated to reflect advancements in knowledge, understanding, care,
clinical trials, and approvals. PPMD also takes a lead role in
advocating for national newborn screening efforts for Duchenne,
Becker, and carrier identification, as well as policy efforts
around access and reimbursement.
ABOUT PARENT PROJECT MUSCULAR DYSTROPHY
Duchenne is a genetic disorder that slowly robs people of
their muscle strength. Parent Project Muscular Dystrophy (PPMD)
fights every single battle necessary to end Duchenne.
We demand optimal care standards and ensure every family has
access to expert healthcare providers, cutting edge treatments, and
a community of support. We invest deeply in treatments for this
generation of Duchenne patients and in research that will benefit
future generations. Our advocacy efforts have secured hundreds of
millions of dollars in funding and won eight FDA approvals.
Everything we do—and everything we have done since our founding
in 1994—helps those with Duchenne live longer, stronger lives. We
will not rest until we end Duchenne for every single person
affected by the disease. Join our fight against Duchenne at
EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and
YouTube.
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SOURCE Parent Project Muscular Dystrophy (PPMD)