Pfizer's mini-dystrophin gene therapy fordadistrogene
movaparvovec failure in the Duchenne muscular dystrophy treatment
opens new opportunities for several pharmaceutical companies such
as Sarepta Therapeutics (SRP-5051), Santhera
Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho
Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to
advance their therapies and potentially dominate the DMD
market.
LAS
VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne
muscular dystrophy is the most prevalent type of muscular dystrophy
found in children. This genetic condition leads to ongoing muscle
weakening and degeneration. It is one among nine different forms of
muscular dystrophy.
According to DelveInsight's estimates, the US accounted for
around ~17K total prevalent cases of Duchenne muscular
dystrophy in 2023. The highest cases were found in the children of
the 5-9 age group, these numbers are expected to grow by the
year 2034.
As per DelveInsight's analysis, the most commonly associated
comorbidities accompanied with DMD included the highest cases
of scoliosis accounting for around ~20% of cases followed by
ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting
for around ~19%, ~18%, and ~15% cases respectively in the US
in the year 2023.
The standard of care for DMD typically involves a combination of
corticosteroids and surgical interventions. Prednisone and
deflazacort are commonly prescribed glucocorticoids that have been
utilized for over two decades to increase muscular strength in DMD
patients. Surgery may be necessary to address contractures, spinal
curvature, or scoliosis, which can affect breathing and overall
mobility. Additionally, interventions such as pacemakers or other
cardiac devices may be utilized to improve heart function.
Sarepta Therapeutics is a key player in the DMD
market, boasting three approved assets and two in the current
pipeline. Despite its dominance, emerging competition is evident.
The historical success rate for market entry varies across the 7MM.
Notably, the DMD market exhibits geographic disparities in
regulatory approvals, with instances of products approved in one
jurisdiction and rejected in another, despite identical clinical
data, reflecting the distinct standards of regulatory bodies such
as EMA and FDA.
Learn more about the FDA-approved DMD drugs @ Drugs
for Duchenne Muscular Dystrophy Treatment
The current US DMD treatment market possesses approved products,
EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51
(eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen),
ELEVIDYS (delandistrogene moxeparvovec) in patients with
DMD.
In January 2024, Santhera
Pharmaceuticals announced the introduction of its AGAMREE
(vamorolone) to treat DMD patients aged four years and above in
Germany. Santhera Pharmaceuticals
has officially entered the commercial stage of its biopharma
journey with the launch of this drug in Germany.
ELEVIDYS, the first FDA-approved gene therapy for DMD, received
accelerated approval in June 2023.
However, ICER recently challenged the potential conversion of
Sarepta's gene therapy to full approval, citing concerns about the
surrogate endpoint used in the approval process.
In the EU4 and the UK, the current market is dominated by
steroid therapies along with an approved medication targeting DMD
patients with the nonsense mutation, TRANSLARNA (ataluren).
In Japan, the only approved
treatment is VILTEPSO (viltolarsen).
ELEVIDYS is a prescription gene therapy designed for
ambulatory children aged 4 to 5 years with DMD and a confirmed
dystrophin gene mutation. It received accelerated approval.
Patients will take oral corticosteroids before and after receiving
ELEVIDYS and will have weekly blood tests to monitor liver enzyme
levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the
first gene therapy for treating pediatric patients aged 4-5 years
with Duchenne Muscular Dystrophy and a confirmed DMD gene
mutation.
VYONDYS 53, developed by Sarepta Therapeutics, is
prescribed for treating DMD in patients with a specific mutation of
the DMD gene that can be addressed by exon 53 skipping. This
approval is under the accelerated approval pathway, based on the
observed increase in dystrophin production in the skeletal muscle
of patients treated with VYONDYS 53. The FDA approved VYONDYS 53
(golodirsen) in December 2019.
Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical
Entity (NCE) exclusivity until December
2024 and Orphan Drug Exclusivity until December 2026.
To know more about DMD treatment options, visit @ New
Treatment for Duchenne Muscular Dystrophy
The DMD market is crowded with so many companies including
FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen
BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics,
Antisense Therapeutics, Capricor Therapeutics, and others are
running clinical trials to improve the treatment space. The DMD
pipeline possesses potential drugs in mid-stage developments to be
launched shortly.
Some of the promising drugs in the pipeline include
Pizuglanstat (TAS-205), SRP-5051, Allogeneic
Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat),
and others that hold the potential to create a significant positive
shift in the DMD market size.
Discover which therapies are expected to grab major
DMD market share @ Duchenne Muscular Dystrophy Market
Report
Italfarmaco is developing Givinostat (ITF2357), an
oral small molecule that functions as a histone deacetylase
inhibitor with potential anti-inflammatory, anti-angiogenic, and
antineoplastic properties. ITF2357 increases the production of
follistatin protein in muscle cells by inhibiting the HDAC enzyme,
leading to increased muscle mass and prevention of muscle
degeneration. This action counters the effects of myostatin,
helping to alleviate the symptoms of DMD. Givinostat is currently
in Phase III trials. The FDA has granted it Orphan Drug, Rare
Pediatric Disease, and Fast Track designations for DMD
treatment. Additionally, the European Commission has designated
Givinostat as an Orphan Medicinal Product for DMD treatment.
TAS-205 (pizuglanstat) is a selective inhibitor of
hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho
Pharmaceutical. It is being advanced as a treatment for Duchenne
muscular dystrophy (DMD), effective irrespective of the type of
dystrophin gene mutation. By inhibiting HPGDS, which worsens the
inflammatory response in the muscles of DMD patients, it helps
control the decline in motor function. Currently, it is in Phase
III of clinical trials.
Discover more about drugs for DMD in development @
Duchenne Muscular Dystrophy Clinical Trials
The anticipated launch of these emerging therapies for Duchenne
muscular dystrophy are poised to transform the market
landscape in the coming years. As these cutting-edge therapies
continue to mature and gain regulatory approval, they are expected
to reshape the Duchenne muscular dystrophy market landscape,
offering new standards of care and unlocking opportunities for
medical innovation and economic growth.
DelveInsight estimates that the market size for DMD is expected
to grow from USD 2.1 billion
in 2023 with a significant CAGR by 2034. The growth of the DMD
market is expected to be mainly driven by increased diagnosed
incidence, patient awareness, and a robust clinical pipeline during
the forecast period (2024–2034).
DelveInsight's latest published market report titled as
Duchenne Muscular Dystrophy Market Insight, Epidemiology, and
Market Forecast – 2034 will help you to discover which
market leader is going to capture the largest market share. The
report provides comprehensive insights into the DMD
country-specific treatment guidelines, patient pool analysis, and
epidemiology forecast to help understand the key opportunities and
assess the market's underlying potential. The DMD market
report proffers epidemiological analysis for the study period
2020–2034 in the 7MM segmented into:
- Total Prevalent Cases of Duchenne Muscular Dystrophy
- Age-specific Cases of Duchenne Muscular Dystrophy
- Ambulatory and Non-ambulatory Cases of Duchenne Muscular
Dystrophy
- Mutation-specific Cases of Duchenne Muscular Dystrophy
- Associated Comorbidities in Duchenne Muscular Dystrophy
The report provides an edge while developing business strategies
by understanding trends shaping and driving the 7MM DMD market.
Highlights include:
- 11-year Forecast
- 7MM Analysis
- Epidemiology-based Market Forecasting
- Historical and Forecasted Market Analysis upto 2034
- Emerging Drug Market Uptake
- Peak Sales Analysis
- Key Cross Competition Analysis
- Industry Expert's Opinion
- Access and Reimbursement
Download this DMD market report to assess the epidemiology
forecasts, understand the patient journeys, know KOLs' opinions
about the upcoming treatment paradigms, and determine the factors
contributing to the shift in the DMD market. Also, stay abreast of
the mitigating factors to improve your market position in the DMD
therapeutic space.
Related Reports
Duchenne Muscular Dystrophy Epidemiology Forecast
Duchenne Muscular Dystrophy Epidemiology Forecast –
2032 report delivers an in-depth understanding of the
disease, historical and forecasted Duchenne muscular
dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy,
France, and the United Kingdom), and Japan.
Duchenne Muscular Dystrophy Pipeline
Duchenne Muscular Dystrophy Pipeline Insight –
2024 report provides comprehensive insights about the
pipeline landscape, pipeline drug profiles, including clinical and
non-clinical stage products, and the key Duchenne muscular
dystrophy companies, including Santhera Pharmaceuticals,
Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd,
FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta
Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid
Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx
Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen,
PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity
Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid
Biosciences Inc, Regenxbio, Stealth BioTherapeutics,
among others.
Nonsense Mutation Duchenne Muscular Dystrophy
Pipeline
Nonsense Mutation Duchenne Muscular Dystrophy Pipeline
Insight – 2024 report provides comprehensive
insights about the pipeline landscape, pipeline drug profiles,
including clinical and non-clinical stage products, and the
key nonsense mutation Duchenne muscular dystrophy companies,
including Taiho Pharmaceutical, Solid Biosciences, Capricor,
Nippon Shinyaku, among others.
Becker Muscular Dystrophy Pipeline
Becker Muscular Dystrophy Pipeline Insight –
2024 report provides comprehensive insights about the
pipeline landscape, pipeline drug profiles, including clinical and
non-clinical stage products, and the key becker muscular
dystrophy companies, including Epirium Bio, Ultragenyx,
Strykagen, among others.
About DelveInsight
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SOURCE DelveInsight Business Research, LLP