|
Forecast Period
|
2025-2029
|
|
Market Size (2023)
|
USD 689.01 Million
|
|
Market Size (2029)
|
USD 963.13 Million
|
|
CAGR (2024-2029)
|
5.71%
|
|
Fastest Growing Segment
|
Stem Cell Therapy
|
|
Largest Market
|
North America
|
Market Overview
Global Amyotrophic Lateral Sclerosis
Therapeutics Market was valued at USD 689.01 Million in 2023 and is expected to
reach USD 963.13 Million by 2029 with a CAGR of 5.71% during the forecast period.
The Global Amyotrophic Lateral Sclerosis
(ALS) Therapeutics Market is evolving rapidly due to rising demand for
innovative treatments addressing this progressive neurodegenerative disease.
ALS primarily affects nerve cells in the brain and spinal cord, leading to loss
of muscle control and mobility. With an increasing global prevalence, the
market is underpinned by growing investments in research and development for
disease-modifying therapies and improved diagnostic methods. Pharmaceutical
companies are focusing on developing advanced therapeutic approaches, including
gene therapy, stem cell therapy, and neuroprotective drugs, which aim to slow
disease progression and improve patient outcomes. Expanding access to
healthcare services and advancements in diagnostic technology are also
contributing to earlier detection and better disease management, boosting
market growth.
Key drivers for the ALS therapeutics
market include the growing awareness of neurodegenerative diseases and the
development of precision medicine targeting ALS-specific pathways. Advocacy
groups and healthcare organizations are playing a vital role in increasing
public understanding of ALS symptoms and treatments, leading to a higher
diagnosis rate and more proactive management. Collaborative efforts between
academic institutions and biotech firms are accelerating the discovery of new
drugs and therapies. Innovations such as the use of biomarkers for personalized
treatment and targeted drug delivery systems are providing promising
opportunities for further expansion of the market.
Despite this progress, the ALS
therapeutics market faces significant challenges. High costs associated with
drug development, stringent regulatory requirements, and the limited
availability of effective treatments remain major hurdles. Additionally, the complexity
of ALS as a disease, with its diverse underlying mechanisms, poses difficulties
in developing universal therapies. Access to advanced care and treatment
options is still limited in many regions, further impeding the market’s growth.
Overcoming these challenges through increased funding, research collaborations,
and technological advancements will be critical in ensuring sustained progress
in the ALS therapeutics landscape.
Key Market Drivers
Rising
Prevalence of ALS and Neurodegenerative Disorders
The rising prevalence of amyotrophic
lateral sclerosis (ALS) and neurodegenerative disorders is a critical driver
for the market's growth. ALS, a progressive disorder affecting nerve cells in
the brain and spinal cord, impacts motor function and leads to muscle degeneration.
For instance, as per the National Institutes of Health (NIH), the
global prevalence of amyotrophic lateral sclerosis (ALS) is projected to rise
significantly, increasing from 222,801 cases in 2015 to 376,674 cases by 2040,
marking a 69% growth. The most substantial regional growth is anticipated in
Africa, with a rise of 116%, followed by Asia at 81% and South America at 73%.
In addition, as per Centers for Disease
Control and Prevention (CDC) 2023 report, the prevalence of amyotrophic lateral
sclerosis (ALS) in the United States stands at approximately 9.1 cases per
100,000 individuals.
Studies also show that aging is a major
risk factor for ALS, aligning with projections that the elderly population will
double by 2050 according to WHO data. This demographic trend is coupled with
improved diagnostic capabilities, allowing for earlier and more accurate
detection of ALS and similar conditions.
Increased funding and collaboration
among stakeholders in healthcare are further propelling the development of
innovative solutions. With neurodegenerative disorders becoming a growing
health burden, the focus on discovering effective treatments continues to shape
advancements in the ALS therapeutics market.
Advancements
in Therapeutic Approaches
Advancements in therapeutic approaches are one of the
key drivers of the growth of the Global Amyotrophic Lateral Sclerosis (ALS)
Therapeutics Market. Traditional treatments for ALS have focused primarily on
managing symptoms and providing palliative care, but recent breakthroughs in
the development of disease-modifying therapies are revolutionizing the
landscape. Gene therapies, which aim to address genetic mutations directly
responsible for ALS, are showing promising results in clinical trials. A notable
example is the development of antisense oligonucleotides (ASOs), such as
nusinersen (Spinraza), which targets genetic mutations that lead to ALS and is
designed to slow the progression of the disease.
Stem cell therapies are also gaining attention as a
potential solution to repair damaged neurons and regenerate lost motor
function. These therapies aim to replace or protect motor neurons affected by
ALS, offering hope for long-term survival and improved quality of life for
patients. In addition to gene and stem cell therapies, other neuroprotective
treatments that target oxidative stress, mitochondrial dysfunction, and
neuroinflammation are being researched, aiming to slow disease progression.
These innovations are being accelerated by
collaboration between biotechnology companies, research institutions, and
patient advocacy groups, which have increased funding and support for ALS
research. As these new therapies progress through clinical trials and
regulatory approvals, they are expected to offer more effective treatment
options and contribute to the market’s rapid growth.
Focus
on Personalized Medicine
The focus on personalized medicine has become a
significant driver for the Global Amyotrophic Lateral Sclerosis (ALS)
Therapeutics Market. Personalized medicine leverages genetic profiling and
patient-specific biomarkers to design targeted treatments that are more
effective and cause fewer side effects. In ALS, where patients experience
varied progression and symptoms, a one-size-fits-all approach is often
inadequate. Personalized treatments allow for more tailored therapies,
addressing the unique genetic factors contributing to ALS in each patient. For
instance, gene therapies that target specific genetic mutations such as those
found in familial ALS provide more precise treatment options compared to
traditional methods. This precision approach has led to the development of
therapies that can slow disease progression and improve outcomes for patients.
The rise in genomic medicine and advancements in
diagnostic tools have facilitated the shift toward personalized ALS treatments.
With the identification of biomarkers that indicate disease progression,
clinicians can tailor treatment plans to individual needs, improving both the
quality of life and survival rates of ALS patients. This move towards
personalized therapies has garnered support from the scientific community and
regulatory bodies, accelerating the approval of treatments. As personalized
medicine continues to gain traction, it has the potential to revolutionize the
ALS treatment landscape, offering hope for more effective management of the
disease.
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Key Market Challenges
High Development
and Treatment Costs
The high development and treatment costs
are significant barriers to the growth of the Global Amyotrophic Lateral
Sclerosis (ALS) Therapeutics Market. ALS is a complex neurodegenerative
disease, and developing treatments for it involves substantial financial
investments. From the discovery phase to clinical trials and final product
commercialization, the process is both lengthy and costly. Research into ALS
treatments often requires the development of novel drug delivery systems,
genetic therapies, and neuroprotective agents, all of which demand advanced
technology, sophisticated infrastructure, and highly specialized expertise. The
expense of these efforts significantly drives up the costs for developing new
therapies.
The clinical trial process is
particularly expensive. ALS, being a rare disease, has a limited patient pool,
which complicates the recruitment for clinical trials. The small number of
patients means that trials are often smaller in scale, which makes it difficult
to obtain statistically significant results. This issue increases both the
duration and cost of trials. Additionally, due to the rarity of ALS, finding
patients who fit the specific trial criteria further exacerbates recruitment
challenges. As a result, pharmaceutical companies often face higher costs per
participant, extending the time required to gain regulatory approval for new
treatments.
Once therapies reach the market, the
cost of treatment remains high. Due to the high cost of research and
development, manufacturers often price new ALS drugs at a premium, making them
inaccessible to many patients. While some insurance plans may cover these
treatments, access can still be limited, particularly in lower-income regions.
The disparity in access to care and therapies further compounds the challenges
faced by patients with ALS. This issue leads to healthcare inequities and slows
the broader adoption of innovative treatments.
Side
effects associated with treatment
One of the significant challenges in the
Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market is the side
effects associated with available treatments. Many current therapies for ALS,
including riluzole and edaravone, are primarily aimed at slowing the disease's
progression rather than offering a cure, and their efficacy can be limited.
These treatments are often associated with various side effects that can hinder
their use in patients. For instance, riluzole has been linked to liver toxicity,
gastrointestinal issues, and fatigue, while edaravone has shown side effects
such as difficulty breathing, skin irritation, and infusion reactions. The
presence of such side effects may discourage patients from adhering to their
treatment regimen, leading to suboptimal therapeutic outcomes.
The nature of ALS as a complex and
progressive disease further complicates treatment, as patients often experience
a combination of symptoms that can exacerbate side effects. For example,
patients with compromised respiratory function may struggle with medications
that impact breathing, while those with weakened muscles may find it difficult
to tolerate treatments that require intravenous administration or other complex
delivery methods. The combination of ALS-related symptoms and treatment side
effects increases the burden on patients and their caregivers, which may affect
their quality of life.
In addition to the direct side effects,
the lack of long-term safety data for some ALS drugs adds another layer of
complexity. Because many of these treatments are still in the clinical or early
approval stages, the long-term consequences and side effect profiles are not
fully understood, creating uncertainty for both physicians and patients. As ALS
is a progressive disease, patients often experience prolonged periods of
treatment, which means the cumulative effects of side effects over time can
become more pronounced, further complicating treatment decisions.
Key Market Trends
Rising
Clinical Trials and Drug Developments
The global Amyotrophic Lateral Sclerosis (ALS)
therapeutics market is experiencing significant momentum, particularly driven
by the surge in clinical trials and drug development efforts aimed at
addressing the unmet needs of ALS patients. With the increasing focus on
understanding ALS's molecular underpinnings, researchers and pharmaceutical
companies are dedicating resources to creating targeted therapies that go
beyond symptom management. Recent advancements in gene therapy, antisense
oligonucleotides, and small molecules are central to this shift. Clinical
trials for new ALS drugs have accelerated, with studies targeting specific
mutations like SOD1, C9orf72, and FUS, which are implicated in familial ALS.
For instance, Ionis Pharmaceuticals’ antisense oligonucleotide, targeting SOD1,
has shown promise in early trials, demonstrating the potential for
gene-specific therapies that could slow disease progression. The success of
such treatments marks a new phase in ALS research, where rather than just managing
symptoms, therapies aim to modify the course of the disease. The FDA's recent
approval of drugs like Radicava and its fast-tracking of ALS-related therapies
further illustrates the commitment to bringing effective treatments to market
quickly. Moreover, the increasing investment in clinical trials is a positive
indicator for the future of ALS therapeutics, with new drugs and treatment
strategies being explored across various phases of clinical development. These
innovations are expected to offer hope for patients, potentially changing the
ALS therapeutic landscape by providing long-term disease-modifying options.
This trend toward growing clinical trials and drug
developments is crucial to the evolution of ALS treatment options. With an
increasing number of trials exploring novel compounds, therapeutic classes, and
combination therapies, there is optimism that these efforts will yield
breakthroughs. ALS, a progressive and fatal neurodegenerative disease, has long
had limited treatment options, making the success of clinical trials an
exciting prospect for patients and the healthcare community. In the coming years,
the landscape of ALS therapeutics could shift dramatically, driven by the
introduction of more targeted and personalized treatment options. These
developments highlight the importance of continued investment in ALS research,
aiming to improve the quality of life for patients and extend survival rates.
Growth
in Personalized and Targeted Therapies
The trend of growth in personalized and targeted
therapies is transforming the global Amyotrophic Lateral Sclerosis (ALS)
therapeutics market. ALS is a complex and progressive neurodegenerative disease
that manifests in various forms, leading to the development of therapies
specifically designed for distinct genetic and molecular subtypes of the
disease. Personalized medicine, which tailors treatment based on an
individual’s genetic makeup, is gaining prominence as it offers the potential
for more effective and precise treatments. This approach is particularly
important in ALS, where the disease may be driven by specific genetic
mutations, such as those seen in familial ALS (FALS).
Targeted therapies are being developed to address the
underlying causes of ALS at the molecular level. For instance, treatments like
antisense oligonucleotides (ASOs) are being researched to target and alter the
genetic instructions of mutated genes that cause ALS. These therapies aim to
slow the progression of the disease and extend patient survival by targeting
specific genes such as C9orf72, SOD1, and TDP-43, which are implicated in ALS.
By focusing on the genetic mutations responsible for ALS, targeted therapies
hold promise in improving outcomes for patients who otherwise have limited
treatment options.
This shift towards personalized medicine and targeted
therapies is spurred by advances in genomic research and technology, as well as
the growing understanding of ALS at the molecular level. The approval of
treatments like nusinersen (Spinraza) and radicava (edaravone), both of which
focus on slowing disease progression, represents a milestone in the evolution
of ALS treatment. These therapies are designed with specific molecular targets
in mind, showcasing the growing trend toward individualized treatment plans.
Moreover, collaborations between biotechnology
companies and research institutions are accelerating the development of new
targeted therapies for ALS. Clinical trials focusing on gene therapies and
other personalized treatment modalities are on the rise, signaling a promising
future for the ALS therapeutics market. As these treatments become more refined
and accessible, they are expected to improve the quality of life for ALS
patients and extend their survival, driving the market forward.
Segmental Insights
Treatment
Insights
In 2023, based on the treatment, the medication
segment is dominating the Global Amyotrophic Lateral Sclerosis Therapeutics
Market. This is due to the increasing development and approval of drug therapies aimed
at slowing the progression of the disease. ALS is a devastating
neurodegenerative condition with no known cure, making effective drug
treatments essential for managing symptoms and prolonging survival. Recent
advancements in medication for ALS, such as edaravone (Radicava) and nusinersen
(Spinraza), have significantly contributed to the growth of this segment. These
drugs, designed to address specific molecular and genetic factors involved in
ALS, offer hope by slowing disease progression, enhancing patient quality of
life, and extending survival.
Furthermore, the growing focus on molecular and
genetic research has led to the development of targeted therapies, including
gene therapy, which continues to make strides in clinical trials. These
therapies are designed to address the root causes of ALS, which have spurred an
increasing demand for innovative medications. With limited treatment options,
ALS medications are often the primary therapeutic approach, making this segment
a critical driver in the market's growth. As a result, the ALS medication segment
is expected to maintain its dominant position in the global market throughout
the forecast period.
Type
Insights
Based on the type, the Sporadic ALS segment
dominated the Global Amyotrophic Lateral Sclerosis Therapeutics Market. This
is due
to the higher incidence and prevalence of this form compared to familial ALS.
Sporadic ALS accounts for approximately 90-95% of all ALS cases worldwide. It
occurs without a clear genetic link and typically manifests in individuals aged
40-70, with men being more commonly affected. The lack of a known genetic cause
makes treatment development challenging but also emphasizes the need for
targeted therapies that focus on slowing disease progression in a broader
patient population.
This widespread occurrence of sporadic ALS contributes
to its market dominance, as pharmaceutical companies are heavily focused on
finding effective treatments for this type, given its higher patient volume.
Moreover, the research and clinical trials related to sporadic ALS have
advanced significantly, with several drugs either in development or already
approved for treating symptoms and slowing disease progression, such as
edaravone (Radicava) and riluzole. These factors drive both research investment
and market growth.
Due to the larger patient base, healthcare providers
are increasingly investing in ALS therapeutics that specifically address
sporadic cases. As a result, there is greater emphasis on therapeutic options
that can be used across a wide patient demographic, including drugs aimed at
reducing oxidative stress, inflammation, and neurodegeneration—all of which are
key factors in sporadic ALS pathophysiology. The market is expected to see
further growth as clinical research continues to refine treatments for sporadic
ALS, providing new opportunities to improve patient outcomes and extend
survival rates.
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Regional Insights
In 2023,
North America emerged as the dominant region in the Global Amyotrophic Lateral
Sclerosis Therapeutics Market, holding the largest market share. This dominance
can be attributed to several key factors, including advanced healthcare
infrastructure, a strong focus on ALS research, and significant investment in
innovative therapeutics. The United States, in particular, stands at the
forefront of the ALS therapeutic market due to its established biotechnology
and pharmaceutical industries, which are actively involved in the development
of cutting-edge treatments. The U.S. government, through organizations like the
National Institutes of Health (NIH), has significantly funded ALS research,
spurring the development of novel therapies.
The
approval of new ALS treatments such as Radicava (edaravone) and Nusinersen
(Spinraza) in North America has further strengthened the region's market
position. The U.S. Food and Drug Administration (FDA) has accelerated the
approval of ALS drugs, facilitating quicker access to promising therapies. As a
result, there has been a steady increase in treatment availability, leading to
better disease management and an improvement in patients' quality of life.
In
addition to regulatory advancements, the region benefits from a growing
awareness of ALS, supported by advocacy groups like the ALS Association. These
organizations have played a critical role in raising public awareness and
driving research funding. Furthermore, North America is home to a large number
of clinical trials for ALS therapeutics, providing a solid foundation for the
development and commercialization of new treatments.
Recent Developments
- In June 2024, Mitsubishi Tanabe Pharma
Corporation (MTPC), part of the Mitsubishi Chemical Group, announced an
agreement with Ethypharm for the transfer of its selective antithrombin agent,
argatroban monohydrate, business in Europe. This move aligns with MTPC's
strategic focus on Radicava (edaravone), a treatment for amyotrophic lateral
sclerosis (ALS), and the company’s decision to prioritize its European
operations around this therapy. The transfer includes the marketing and sales
of argatroban in the UK, Germany, and other European markets, which were
previously managed by MTPC’s subsidiaries Mitsubishi Tanabe Pharma Europe Ltd
(MTPE) and Mitsubishi Tanabe Pharma GmbH (MTPD).
- In February 2024, Biogen Inc. announced
that the Committee for Medicinal Products for Human Use (CHMP) of the European
Medicines Agency (EMA) issued a positive opinion recommending marketing
authorization under exceptional circumstances for QALSODY (tofersen) for adults
with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide
dismutase 1 (SOD1) gene. If approved by the European Commission, QALSODY will
become the first treatment in the European Union targeting a genetic cause of
ALS, also known as motor neuron disease (MND).
- In January 2023, Amylyx Pharmaceuticals
entered into an exclusive license and distribution agreement with Neopharm to
commercialize AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) for ALS
treatment in Israel, Gaza, West Bank, and the Palestinian Authority, pending
regulatory approval. This agreement supports Amylyx's expansion efforts in the
EMEA region and aims to increase access to AMX0035 for ALS patients. This partnering
with Neopharm would help accelerate access to treatment for eligible ALS patients
by leveraging potential approvals from other regions in the Israel.
Key Market Players
- Mitsubishi
Tanabe Pharma Corporation
- Biogen
Inc.
- Brainstorm
Cell Therapeutics Ltd.
- Amylyx
Pharmaceuticals, Inc.
- Ionis
Pharmaceuticals, Inc.
- Sanofi
S.A.
- Otsuka
Pharmaceutical Co., Ltd.
- Sun Pharmaceutical
Industries Ltd.
- Orion
Corporation
- AB
Science
|
By Treatment
|
By Type
|
By Distribution Channel
|
By Region
|
- Medication
- Stem Cell Therapy
- Others
|
|
- Hospital Pharmacies
- Retail Pharmacies
- Others
|
- North America
- Europe
- Asia Pacific
- South America
- Middle East & Africa
|
Report Scope:
In this report, the Global Amyotrophic
Lateral Sclerosis Therapeutics Market has been segmented into the following
categories, in addition to the industry trends which have also been detailed
below:
- Amyotrophic
Lateral Sclerosis Therapeutics Market, By Treatment:
o
Medication
§
Riluzole
§
Edaravone
§
Others
o
Stem
Cell Therapy
o
Others
- Amyotrophic
Lateral Sclerosis Therapeutics Market, By Type:
o
Sporadic
ALS
o
Familial
ALS
- Amyotrophic
Lateral Sclerosis Therapeutics Market, By Distribution Channel:
o
Hospital
Pharmacies
o
Retail
Pharmacies
o
Others
- Amyotrophic
Lateral Sclerosis Therapeutics Market, By Region:
o
North
America
§
United
States
§
Canada
§
Mexico
o
Europe
§
France
§
United
Kingdom
§
Italy
§
Germany
§
Spain
o
Asia-Pacific
§
China
§
India
§
Japan
§
Australia
§
South
Korea
o
South
America
§
Brazil
§
Argentina
§
Colombia
o
Middle
East & Africa
§
South
Africa
§
Saudi
Arabia
§
UAE
Competitive Landscape
Company
Profiles: Detailed
analysis of the major companies present in the Global Amyotrophic Lateral
Sclerosis Therapeutics Market.
Available Customizations:
Global Amyotrophic Lateral Sclerosis Therapeutics Market
report with the given
market data, TechSci Research offers customizations according to a company's
specific needs. The following customization options are available for the
report:
Company Information
- Detailed analysis and profiling of additional market players (up to
five).
Global Amyotrophic Lateral Sclerosis Therapeutics Market is an upcoming report to be released
soon. If you wish an early delivery of this report or want to confirm the date
of release, please contact us at [email protected]