US FDA approves Sarepta's gene therapy for rare muscular dystrophy in
some kids
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[June 23, 2023]
By Leroy Leo and Aditya Samal
(Reuters) - The U.S. health regulator has granted accelerated approval
to Sarepta Therapeutics' first-of-its-kind gene therapy for Duchenne
muscular dystrophy (DMD), an inherited progressive muscle-wasting
disorder that almost always affects young boys.
Sarepta said on Thursday the Food and Drug Administration had approved
the treatment for children aged between 4 and 5 years who can walk. It
was initially seeking approval for all DMD patients who can walk.
The therapy's wholesale acquisition cost is $3.2 million, CEO Douglas
Ingram said on a conference call with investors.
The world's most expensive treatment, a gene therapy for bleeding
disorder hemophilia from Australian drugmaker CSL Ltd, carries a list
price of $3.5 million.
The agency's decision comes after several delays and questions over the
effectiveness of the therapy, which will be sold under the brand name
Elevidys.
Elevidys could change the way that DMD patients, who rarely survive
beyond their thirties, are treated. Currently, approved steroids and
some therapies for specific genetic mutations only control symptoms and
slow disease progression.
![](http://archives.lincolndailynews.com/2023/Jun/23/images/ads/current/graue_sda_042021.png)
In a mid-stage trial, Sarepta's gene therapy was able to produce a mini
version of the dystrophin protein needed to help keep muscles intact,
but did not improve patient clinical outcomes like their ability to walk
and stand.
A late-stage study to confirm the treatment's effectiveness is underway
and initial data from it is expected by December.
Current options for DMD patients are limited. Of four new "exon-skipping"
therapies - intended for a smaller group of patients with specific
genetic mutations - three are from Sarepta.
They require weekly infusions and work by skipping specific parts of
genes, called exons, with the aim of allowing the body to form shorter
forms of the dystrophin protein. Elevidys should not be used in patients
with any deletion in exon 8 and exon 9 in the DMD gene, the company said
on Thursday.
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Signage is seen outside of the Food and
Drug Administration (FDA) headquarters in White Oak, Maryland, U.S.,
August 29, 2020. REUTERS/Andrew Kelly/File Photo
![](http://archives.lincolndailynews.com/2023/Jun/23/images/ads/current/bees_lda_HEROES_2023.png) Sarepta's infrastructure is prepared
to support the launch of Elevidys and it could take about four
months to get to patients even with a limited target population,
Ingram said ahead of the approval.
DMD affects an estimated one-in-3,500 male births worldwide,
according to the National Organization for Rare Disorders.
"We will have a very successful launch and I think we'll expand this
label successfully early next year," Sarepta's Ingram said.
Elevidys could become a multibillion-dollar product, William Blair
analyst Tim Lugo said in a note.
A green light for Sarepta's treatment could open the doors for more
effective gene therapies, experts and analysts have said. Pfizer is
also developing a gene therapy for DMD.
A late-stage trial of 126 patients is ongoing at Sarepta, and will
be pivotal in expanding the patient population and gaining a
traditional approval.
Roche acquired the commercial rights to Elevidys outside the United
States under a 2019 deal. Sarepta remains in charge of clinical
development, but it splits the costs with Roche.
Sarepta's shares were up nearly 1% in extended trading.
(Reporting by Aditya Samal, Leroy Leo and Raghav Mahobe in Bengaluru;
Editing by Shinjini Ganguli, Maju Samuel and Devika Syamnath)
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