Courier Roche co-developed, DMD gene therapy launched marketing application

▎Editor of WuXi AppTec content team

Recently, Sarepta Therapeutics submitted a Biological Product Licensing Application (BLA) to the U.
S.
FDA to seek accelerated approval for the gene therapy SRP-9001 for the treatment of patients with
Duchenne muscular dystrophy (DMD).
SRP-9001 is an ongoing gene therapy for DMD developed in collaboration with Roche, and in previously published research data it demonstrates its continued efficacy
in patients with DMD.

DMD is a rare and fatal genetic disease, with about 1 in every 3500-5000 male newborns worldwide
.
DMD is an X-linked recessive disease
caused by mutations in the gene encoding dystrophin.
DMD usually occurs in infants and young children, and children may experience developmental delays (eg, difficulty walking, standing from a sitting position
).
As the disease progresses, symptoms of muscle weakness in the lower extremities can spread to the arms and other areas
.
Most patients require full-time wheelchair use by the age of teen and gradually lose the ability to
carry out daily life independently.
Finally, patients begin to rely on ventilation therapy as respiratory muscle dysfunction causes more and more severe breathlessness, and heart failure due to cardiac dysfunction is the most common cause of death in patients with DMD, who usually die
in their 20s.

SRP-9001 (delandistrogene moxeparvovec) is an investigational gene therapy designed to deliver the gene of interest to muscle tissue and specifically produce the active ingredient
of anti-amyotrophin.
In December 2019, Roche partnered with Sarepta to accelerate access to SRP-9001 treatment
for DMD patients outside the United States.
The company aims to seek accelerated approval
for SRP-9001.
The review of accelerated approval will be based on the use of SRP-9001 expressed micro-anti-amyotrophin levels as an alternative endpoint to predict clinical benefit
.
In addition, this BLA application is based on previous positive results
in preclinical studies, biomarker studies, and clinical functional studies.
In clinical trials, SRP-9001 treatment showed positive results at multiple time points, including one, two, and four years after treatment, in addition to consistently safe performance
.
The BLA submission includes efficacy and safety data from multiple clinical studies (SRP-9001-101, SRP-9001-102, SRP-9001-103) and a comprehensive analysis
covering all three studies.

It is understood that Sarepta proposed EMBARK (code SRP-9001-301), a study that has completed the recruitment of subjects, as a post-marketing confirmatory study
to support accelerated approval.
EMBARK is a global randomized, double-blind, placebo-controlled trial whose primary endpoint was to assess the change in the overall North Star Dynamic Assessment (NSAA) score of members of the
SRP-9001 treatment group from baseline to week 52 compared with the placebo control group.

Image Credit: 123RF
"Every moment, the relentless disease of DMD is taking away the muscles of thousands of children, and at the same time it is taking away their future," Dr.
Doug Ingram, president and CEO of Sarepta Therapeutics, said in a press release.
Sarepta's submission of a BLA application for accelerated approval of SRP-9001 is an important milestone
in our long-term commitment to providing emergency therapeutic interventions to children.
If approved, SRP-9001 will be the first gene therapy
available for DMD patients.
We are very grateful to the brave families who participated in the SRP-9001 trial, as well as the clinical researchers and experts who participated in the trial, who provided us with guidance and played a key role
in achieving this milestone.
" "
To date, the RP-9001 has made several regulatory advances
.
It was granted fast-track eligibility by the FDA in July 2020, an approval process by the U.
S.
FDA to facilitate the development and rapid review
of therapies for the treatment of serious diseases and to fill unfinished medical needs.
In addition to fast-track qualification, SRP-9001 has been granted rare pediatric disease (RPD) designation in the United States and orphan drug designation
in the United States, the European Union, Switzerland and Japan.

---

As WuXi AppTec's CTDMO focused on cell and gene therapies, WuXi Biologics is committed to accelerating and transforming the development, testing, production and commercialization
of gene and cell therapies and other high-end therapies.
WuXi Sheng is able to help customers around the world bring more innovative therapies to market early for the benefit of patients
.
If you have relevant business needs, please click on the picture below to fill in the specific information
.

▲If you have any business needs, please long press to scan the QR code above, or