Amyotrophic Lateral Sclerosis Als Market

DelveInsight's "Amyotrophic Lateral Sclerosis- Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the ALS, historical and forecasted epidemiology as well as the ALS market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

 

The ALS market report provides current treatment practices, emerging drugs, ALS market share of the individual therapies, current and forecasted ALS market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current ALS treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.

Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2019-2032

ALS Disease Understanding and Treatment Algorithm

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. The disease is progressive, meaning the symptoms get worse over time. ALS belongs to a wider group of disorders known as motor neuron diseases caused by gradual deterioration (degeneration) and death of motor neurons. Motor neurons are nerve cells that extend from the brain to the spinal cord and muscles throughout the body. Messages from motor neurons in the brain (called upper motor neurons; UMN) are transmitted to motor neurons in the spinal cord and to motor nuclei of the brain (called lower motor neurons; LMN) and from the spinal cord and motor nuclei of the brain to a particular muscle or muscles. ALS can be either sporadic or genetic. The sporadic type is the most common and can affect anyone. The genetic or familial type is rarer. Common symptoms include painless, progressive muscle weakness. The first thing a person might notice is tripping more often, or dropping things because of the weakness. Slurred speech, difficulty swallowing, and trouble breathing can occur.

 

Diagnosis

ALS is a difficult disease to diagnose. There is no one test or procedure to establish the diagnosis of ALS ultimately. It is through a clinical examination and a series of diagnostic tests, often ruling out other diseases that mimic ALS. The diagnosis of ALS relies on medical history, physical examination, electrodiagnostic testing (with needle EMG), and neuroimaging. Biomarkers can play a crucial role in diagnostic, prognostic, or predictive research studies. They could potentially become important for the stratification of patients and monitoring treatment effects in clinical trials. Genetic testing of the five most prevalent genes found to be mutated in ALS is routinely offered to patients with a positive family history (C9orf72, SOD1, TDP‐43, FUS, and TBK‐1).

 

Treatment

There is no cure for ALS, so treatment aims to alleviate symptoms, prevent unnecessary complications, and slow the rate of disease progression. Medical interventions and technology have vastly improved the quality of life for people with ALS by assisting with breathing, nutrition, mobility, and communication. Modern therapeutic strategies comprise of neuroprotective treatment focused on antiglutamatergic, antioxidant, antiapoptotic, and anti-inflammatory molecules. Drugs approved for the treatment of ALS are IV Radicava (Mitsubishi Tanabe Pharma), Rilutek/Riluzole) (Sanofi/Covis), Exservan (Aquestive Therapeutics), and Tiglutik/ Teglutik (ITF Pharma). In ALS, opioids and nonsteroidal anti-inflammatory drugs (NSAIDs) are widely used to relieve discomfort. A combination of quinidine and dextromethorphan called Nuedexta (Avanir Pharmaceuticals) has been approved by the US Food and Drug Administration to treat pseudobulbar effect, however it can lengthen the QT interval, raising the risk of cardiac arrhythmia.

ALS Epidemiology

The ALS epidemiology division provides insights about historical and current ALS patient pool and forecasted trends for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

 

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted ALS epidemiology [segmented as total prevalent cases, total diagnosed cases, age-specific cases, type-specific cases, mutation-specific cases, site of onset and gender-specific cases of ALS] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country Wise- ALS Epidemiology

  • Estimates show that the highest cases of ALS in the 7MM were in the United States, followed by Japan, Germany, the United Kingdom, Italy, France, and Spain in the year 2021.
  • The total diagnosed prevalent cases of ALS in the 7MM range from 52,099 in 2021 growing at a CAGR of 1.04% during the study period (2019–2032).
  • Epidemiology assessed for the condition showed that the US, in 2021, accounted for approximately 20,556 diagnosed prevalent cases of ALS.
  • Among the EU-5 countries in 2021, Germany had the highest diagnosed prevalent cases of ALS with 5,061 cases, followed by UK (4,276) and Italy (4,226). In contrast, Spain had the lowest cases (3,065) in 2021.
  • Japan accounted for 10,742 diagnosed prevalent ALS cases in 2021.
  • In the United States, in 2021, the proportion of male and female cases were 11,902 and 8,654, respectively.
  • As per the analysis, a higher percentage of sporadic ALS was observed in the 7MM compared to familial ALS in 2021.
  • ALS can be divided into C9ORF72, SOD1, FUS, others mutations (TARDBP, OPTN, ANG, etc.), and non-mutated/unidentified mutation based on the types of mutations causing the condition. In the United States, the number of cases of C9ORF72, SOD1, FUS, others mutations (TARDBP, OPTN, ANG, etc.), and non-mutated/unidentified mutation was 1,747, 514, 267, 1,743 and  16,284 respectively, in 2021.
  • In 2021, the patients with the spinal site of onset accounted for 12,950 cases, followed by 5,961 cases with the bulbar site of onset, and 1,644 cases with other uncertain regions in the US.
  • Japan accounted for approximately 3,738 cases of 70-79 years of age of ALS in 2021.

ALS Drug Chapters

Drug chapter segment of the ALS report encloses the detailed analysis of ALS marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the ALS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

 

Marketed Drugs

 

Radicava (Mitsubishi Tanabe Pharma Corporation)

Radicava (edaravone) is a free radical scavengers and administered as intravenous infusion of 60 mg over 60 minutes. Radicava received fast track and orphan drug designation by FDA and Orphan drug designation by EMA. It is approved in the USA and Japan for the treatment of ALS. In May 2019, Radicava, Mitsubishi Tanabe Pharma notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Radicava intended for the treatment of ALS. The company is currently conducting a Phase III trial for long-term safety and tolerability of oral edaravone for the treatment of ALS.

 

Exservan (Aquestive Therapeutics)

Exservan (Riluzole oral film) consists of a thin film that is placed on the tongue, utilizes the company’s “PharmFilm” technology. The dissolving oral film can be taken twice daily without water, making it easier for patients who have difficulty swallowing pills or liquids. Riluzole oral film received the US FDA Orphan Drug designation which is used as adjunctive therapy in the treatment of ALS. Aquestive Therapeutics announced the exclusive license to Mitsubishi Tanabe Pharma Holdings America (MTHA) for the commercialization Exservan in the United States and Zambon for the development and commercialization of Exservan in the European Union for the treatment of ALS.

 

Tiglutik/ Teglutik (ITF Pharma)

Tiglutik (riluzole) oral suspension is indicated for the treatment of ALS. Tiglutik is the first and only easy-to-swallow thickened riluzole liquid for ALS and is administered twice daily via an oral syringe. Teglutik was first launched in the European markets after it was approved by US FDA as Tiglutik for the treatment of ALS.

 

Nuedexta (Avanir Pharmaceuticals)

Nuedexta (dextromethorphan hydrobromide/quinidine sulfate) is the only approved medication that has proven to be effective in lowering down the Pseudobulbar Affect (PBA). Nuedexta acts on sigma-1 and NMDA receptors in the brain, although the mechanism by which Nuedexta exerts therapeutic effects in patients with PBA is unknown. It is approved in the USA and Europe for the treatment of PBA associated with certain neurological conditions such as ALS.

Note: Detailed Current therapies assessment will be provided in the full report of ALS

 

ALS Emerging Drugs

 

AMX0035 (Amylyx Pharmaceuticals)

Amylyx Pharmaceuticals is developing AMX0035, an investigational neuroprotective therapy, which is a fixed-dose co-formulation of two active compounds, namely, sodium phenylbutyrate (PB) and Taurursodiol (tauroursodeoxycholic acid [TUDCA]). Based on data from the Phase II CENTAUR trial, the FDA has accepted Amylyx‘s NDA for AMX0035 for the treatment of ALS. The FDA also granted Priority Review and assigned a prescription drug user fee act date for AMX0035 of June 29, 2022. A phase III (Phoenix) trial of the drugs is currently ongoing. AMX0035 has the potential to become the newest approval for the treatment of ALS. We expect this drug to compete with Oral Radicava, and other drugs from the HEALEY ALS platform trial. Although Amylyx is expected to enter the US and EU5 market first when compared to Oral Radicava, which is expected to follow the footsteps of IV Radicava in terms of first market entry in Japan.

 

Tofersen (Biogen/Ionis Pharmaceuticals)

Tofersen (Biogen/Ionis Pharmaceuticals), also known as BIIB067, is an antisense drug designed to reduce the production of superoxide dismutase 1 (SOD1), which is the best understood genetic cause of familial ALS. The drug is being currently investigated in two Phase III trials for the treatment of ALS caused by SOD1 mutation. In October 2021, the company presented the Phase III results from VALOR (Part C), which showed that the trial did not meet the primary endpoint. This failure could likely hinder Biogen’s planned filing for FDA approval in the ALS market.

 

ION363 (Ionis Pharmaceuticals)

Ionis Pharmaceuticals’ portfolio for ALS also includes ION363, another investigational antisense medicine for ALS, designed to reduce the Fused in Sarcoma (FUS) protein production. The drug is owned by Ionis and is in development for patients with a rare genetic form of ALS caused by mutations in the FUS gene, which causes motor neuron degeneration through a toxic gain of function mechanism. ION363 can potentially reduce or prevent disease progression in FUS-ALS patients, and the data from the ongoing Phase III trial is expected in 2024.

 

Masitinib (AB Sciences)

AB Sciences’ Masitinib is an orally administered tyrosine kinase inhibitor, which has already completed a Phase II/III trial and has attained positive results, followed by a green signal from the US FDA on the IND application. The drug is under investigation for a Phase III trial in patients with ALS but was previously put on hold due to a potential risk of ischemic heart disease with masitinib, voluntary by the company – the trial was resumed after the FDA authorization.

 

Verdiperstat (Biohaven)

Biohaven’s Verdiperstat is a first-in-class, potent, selective, brain-permeable, irreversible myeloperoxidase (MPO) enzyme inhibitor. The drug was licensed from AstraZeneca in 2018. It was selected as an investigational therapy for inclusion in the first HEALEY ALS platform trial by an independent scientific advisory committee in September 2019 and is currently in Phase II/III clinical trial for the treatment of ALS, for which top-line results are anticipated in mid-2022. The drug has recently failed in a Phase III trial of Multiple System Atrophy, thereby disappointing our hopes for ALS till the time any convincing results are out for ALS specifically.

Note: Detailed emerging therapies assessment will be provided in the final report.

ALS Market Outlook

Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases mainly involving the nerve cells (neurons) responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time.

 

Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. The treatment landscape of ALS includes multidisciplinary care, such as physical therapy, speech therapy, dietary counselling, heat or whirlpool therapy and others. Medications are also prescribed to help manage symptoms of ALS, including pain, muscle cramps, stiffness, excess saliva and phlegm, and the pseudobulbar affect (involuntary or uncontrollable episodes of crying and/or laughing, or other emotional displays). Drugs also are available to help individuals with pain, depression, sleep disturbances, and constipation.

 

There is no other drug which is globally approved for slowing the progression of ALS. Rilutek, Tiglutik, and Exservan are brand names for different formulations of riluzole, a medicine used in the treatment of ALS. Rilutek is an oral tablet, Tiglutik is an oral suspension, and Exservan is an oral film. Neudexta is approved for the treatment of pseudobulbar effects in conditions such as multiple sclerosis and ALS. Mitsubishi Tanabe Pharma received approval to market Radicut (edaravone or MCI-186) for ALS treatment, strictly limited to Japan. Radicava was approved to treat ALS in the US in May 2017, the first new ALS treatment agent in the US in 22 years.

 

Nevertheless, recently some developmental initiatives have been taken towards the treatment of ALS. Several pharmaceutical key players have taken to the initiative to meet the unmet needs of the present situation of the ALS market. Some of the key players are in the late and mid clinical development stages with their leading drug candidates.

 

The ALS pipeline possesses drugs in the late-stage development, many of which hold the potential to get launched in the forecast period. The key players include AMX0035 (Amylyx Pharmaceuticals), Tofersen/BIIB067 (Biogen/Ionis Pharmaceuticals), ION363 (Ionis Pharmaceuticals), Ibudilast (MediciNova), Masitinib (AB Science), Pegcetacoplan (Apellis Pharmaceuticals), NurOwn (Brainstorm-Cell Therapeutics), Reldesemtiv (Cytokinetics), Gold Nanocrystals/CNM-Au8 (Clene Nanomedicine), Verdiperstat (Biohaven Pharmaceuticals), Zilucoplan (UCB Pharma), and others investigating their candidates for the treatment of ALS in the 7MM.

 

According to DelveInsight, ALS market in 7MM is expected to witness a major change in the study period 2019-2032.

 

Key Findings

  • The market size of ALS in the seven major markets is expected to rise from USD 460.4 million in 2021 during the study period (2019–2032).
  • Current market dynamics are dominated by the use of Rilutek, Tiglutik, Exservan, Neudexta, Radicava and others (Anti-epileptic drugs, Opioids, NSAIDs, Diuretics, SSRIs, Antidepressants, etc.)
  • The therapies with the potential to get launched in the forecast period include AMX0035, Tofersen, ION363, NurOwn, Masitinib, Pegcetacoplan (APL-2) and others. The launch of these therapies may increase market size in the coming years, assisted by an increasing patient pool of ALS patients.
  • The United States accounts for the largest market size of ALS compared to EU5 (Germany, the United Kingdom, Italy, France, and Spain) and Japan.
  • In the United States, the market size of ALS is anticipated to rise from USD 324.6 million in 2021 during the forecast period.
  • Among the EU5 countries, Germany had the largest market size (USD 17.7million) in 2021, while Spain had the smallest with USD 10.7 million.
  • In 2021, Japan accounted for a market size of USD 63.2 million.

 

The United States Market Outlook

The total market size of ALS in the United States is expected to increase with a CAGR of 23.4% in the study period (2019–2032).

 

EU-5 Countries: Market Outlook

The total market size of ALS in EU5 is expected to increase with a CAGR of 30.4% in the study period (2019–2032).

 

Japan Market Outlook

The total market size of ALS in the Japan is expected to increase with a CAGR of 20.9% in the study period (2019–2032).

Analyst Commentary

  • The pipeline of ALS is very robust, many potential therapies are being investigated for the treatment of ALS, and it is safe to predict that the treatment space will experience a significant impact on the market during the forecast period.
  • Most drugs that are in the early stage or have failed to meet their primary endpoints were assumed to have a 50% chance of crossing the line. In the emerging pipeline highest probability of success (POS) was given to AMX0035 75%, followed by Reldesemtiv, and ION363 65%.
  • The European market has been quite uncertain about ALS drugs as Nuedexta was initially authorized by the EMA but later withdrawn at the request of the marketing authorization holder. Same scenario occured with Radicava, as Mitsubishi Tanabe Pharma notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for the treatment of ALS. So, these two therapies are currently not available in the Europe.
  • Although tofersen did not meet the primary endpoint in its Phase III trial, but trends favoring tofersen were seen across multiple secondary and exploratory measures of biologic activity and clinical function. This setback will likely hinder Biogen’s planned filing for FDA approval in the ALS market and actively engaging with regulators, the medical community, patient advocacy groups, and other key stakeholders worldwide to determine potential next steps.

ALS Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the ALS market or expected to get launched in the market during the study period 2019-2032. The analysis covers ALS market uptake by drugs; patient uptake by therapies; and sales of each drug. For example- 

 

Tofersen (Biogen/Ionis Pharmaceuticals), is an antisense drug designed to reduce the production of superoxide dismutase 1 (SOD1) is expected to first launch in USA (2022) followed by EU5 (2023) and Japan (2024). As per our analysis, Tofersen’s drug uptake in 7MM is expected to be fast and with an estimated probability of success to be 50%. As SOD1 cover only 2% cases of total ALS in USA and EU5, so peak share is expected to be 1.3% in USA and EU5, whereas in Japan SOD1 is expected to cover 4% of the total ALS pool, so expected peak share will be 2.5%.

Note: Detailed emerging therapies assessment will be provided in the final report.

ALS Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II and Phase III stage. It also analyses ALS key players involved in developing targeted therapeutics.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing, patent details and other information for ALS emerging therapies.

Reimbursement Scenario in ALS

HealthWell Foundation is an independent non-profit organization providing financial assistance to adults and children to cover the cost of prescription drug coinsurance, copayments, deductibles, health insurance premiums and other selected out-of-pocket health care costs of ALS. It has an ALS fund that provides copay/premium as well as a pharmacy card fund. The treatments for ALS covered under the fund are Botox, Cuvposa, Edaravone, Elavil, Gabapentin, Glycopyrrolate, Increlex, Nuedexta, Radicava, Rilutek, Riluzole, and Tiglutik. The fund provides a maximum award level of USD 15,000 per year.

 

The Patient Advocate Foundation’s (PAF) Copay Relief (CPR) Program also has an ALS fund that provides copay, coinsurance and deductible. The fund has been developed in response to patients who have contacted PAF for help with their medication expenses and failed to receive it. The fund provides a maximum award level of USD 5,000 per year.

 

Besides these organizations, certain companies also provide financial assistance to ALS patients. For instance, ITF Pharma provides support and services for those impacted by ALS. The company’s Tiglutik copay support helps ensure that people get financial assistance in accessing the treatment they need. Eligible patients need not pay more than USD 50 per filled prescription of Tiglutik. The service is valid for each prescription fill for commercially insured patients where Tiglutik is covered.

 

Similarly, Mitsubishi Tanabe Pharma America has an out-of-pocket assistance program to support eligible patients with commercial insurance. Patients who have commercial insurance coverage for treatment with Radicava are eligible for enrollment into the program. With this program, eligible patients can save on the deductible, copay, and coinsurance costs for their medication and infusion costs for Radicava.

KOL- Views

To keep up with current market trends, we take KOLs and SME's opinion working in ALS domain through primary research to fill the data gaps and validate our secondary research. Some of the leaders are Principal Investigator and Center Director, Washington University School of Medicine; Professor of neurology, and Chairman, Department of Neurology, University Hospital and Medical Facility of Ulm; Principal Investigators and Professor of Neurology at Harvard Medical School and the Director of the Healey Center for ALS and Chair of Neurology at Mass General Hospital and others. Their opinion helps to understand and validate current and emerging therapies treatment patterns or ALS market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the ALS Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

  • The report covers the descriptive overview of ALS, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
  • Comprehensive insight has been provided into the ALS epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for ALS are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of ALS market; historical and forecasted is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global ALS market

Report Highlights

  • In the coming years, ALS market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence ALS R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition     
  • Major players are involved in developing therapies for ALS. Launch of emerging therapies will significantly impact the ALS market
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for ALS
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

ALS Report Insights

  • Patient Population
  • Therapeutic Approaches
  • ALS Pipeline Analysis
  • ALS Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

ALS Report Key Strengths

  • 11 Years Forecast
  • 7MM Coverage
  • ALS Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

ALS Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the ALS drug class share (%) distribution in 2019 and how it would look like in 2032?
  • What would be the ALS total market size as well as market size by therapies across the 7MM during the forecast period (2019-2032)?
  • What are the key findings pertaining to the market across 7MM and which country will have the largest ALS market size during the forecast period (2019-2032)?
  • At what CAGR, the ALS market is expected to grow in 7MM during the forecast period (2019-2032)?
  • What would be the ALS market outlook across the 7MM during the forecast period (2019-2032)?
  • What would be the ALS market growth till 2032, and what will be the resultant market Size in the year 2032?
  • How would the unmet needs affect the market dynamics and subsequent analysis of the associated trends?

 

Epidemiology Insights:

  • What is the disease risk, burden and regional/ethnic differences of the ALS?
  • What are the key factors driving the epidemiology trend for seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What is the historical ALS patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of ALS in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • Where will be the growth opportunities in the 7MM with respect to the patient population pertaining to ALS?
  • Out of all 7MM countries, which country would have the highest prevalent population of ALS during the forecast period (2019-2032)?
  • At what CAGR the patient population is expected to grow in 7MM during the forecast period (2019-2032)?

 

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

  • What are the current options for the ALS treatment in addition to the approved therapies?
  • What are the current treatment guidelines for the treatment of ALS in the USA, Europe, and Japan?
  • What are the ALS marketed drugs and their respective MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of ALS?
  • How many therapies are in-development by each company for ALS treatment?
  • How many are emerging therapies in mid-stage, and late stage of development for ALS treatment?
  • What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the ALS therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies being developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for ALS and their status?
  • What are the current challenges faced in drug development?
  • What are the key designations that have been granted for the emerging therapies for ALS?
  • What are the global historical and forecasted market of ALS?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the ALS market
  • To understand the future market competition in the ALS market and Insightful review of the key market drivers and barriers
  • Organize sales and marketing efforts by identifying the best opportunities for ALS in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors
  • Organize sales and marketing efforts by identifying the best opportunities for ALS market
  • To understand the future market competition in the ALS market

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Key Events

5. SWOT Analysis

6. Amyotrophic Lateral Sclerosis (ALS) Market Overview at a Glance

6.1. Market Share (%) Distribution by Therapies in 2022

6.2. Market Share (%) Distribution by Therapies in 2032

7. Disease Background and Overview

7.1. Introduction

7.2. Types of ALS

7.2.1. Sporadic ALS

7.2.2. Genetic or Familial ALS

7.3. Causes

7.3.1. Genetics

7.3.2. Environmental factors

7.4. Risk factors

7.5. Symptoms

7.6. Clinical Features

7.6.1. Clinical presentation

7.6.2. Amyotrophic lateral sclerosis phenotypes

7.6.3. Subtypes of ALS based on relative UMN versus LMN involvement

7.6.4. Subtypes of motor neuron disease based on the regional distribution of involvement

7.6.5. Subtypes of ALS based on additional frontotemporal involvement

7.7. Pathogenesis

7.7.1. Failure of proteostasis

7.7.2. Disturbed RNA metabolism

7.7.3. Cytoskeletal disturbances and axonal transport defects

7.8. Biomarker

7.9. Prediction of prognosis

7.10. Differential Diagnosis

7.11. Diagnosis

7.11.1. Criteria and requirements for diagnosis

8. Treatment and Management of ALS

8.1. Multidisciplinary Care

8.2. Neuroprotective Treatments

8.2.1. Riluzole

8.2.2. Edaravone

8.2.3. Nuedexta

8.3. Symptomatic Treatments

8.4. Adaptive and Assistive Equipment

8.5. Nutrition

8.6. Respiratory Management

8.7. Secretion Management

8.8. Palliative and End-of-Life Care

9. European Federation of Neurological Societies (EFNS) guidelines on the Clinical Management of ALS (MALS) (2012)

10. American Association of Neurology (AAN) Guidelines for Care and Management of patients with ALS (2009)

11. Management Algorithm for Respiratory Dysfunction in ALS

12. Management guidelines for ALS in Japan, the United States, and Europe.

13. Epidemiology and Patient Population of 7MM

13.1. Key Findings

13.2. Epidemiology Methodology

13.3. Total Prevalent Population of Amyotrophic Lateral Sclerosis (ALS) in the 7MM

13.4. Total Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis (ALS) in the 7MM

13.5. Assumptions and Rationale

13.6. The United States

13.6.1. Prevalence of Amyotrophic Lateral Sclerosis in the United States

13.6.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in the United States

13.6.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in the United States

13.6.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in the United States

13.6.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in the United States

13.6.6. Site of onset of Amyotrophic Lateral Sclerosis in the United States

13.6.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in the United States

13.7. EU5

13.7.1. Prevalence of of Amyotrophic Lateral Sclerosis (ALS) in EU5

13.7.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis (ALS) in EU5

13.7.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis (ALS) in EU5

13.7.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis (ALS) in EU5

13.7.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis (ALS) in EU5

13.8. Japan

13.8.1. Prevalence of Amyotrophic Lateral Sclerosis in Japan

13.8.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in Japan

13.8.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in Japan

13.8.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in Japan

13.8.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in Japan

13.8.6. Site of onset of Amyotrophic Lateral Sclerosis in Japan

13.8.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in Japan

14. Organizations contributing towards ALS

15. Patient Journey

16. Marketed Drugs

16.1. Key Competitor

16.2. Radicava (edaravone injection): Mitsubishi Tanabe Pharma Corporation

16.2.1. Drug Description

16.2.2. Regulatory Milestones

16.2.3. Other Developmental Activities

16.2.4. Current Pipeline Activity

16.2.5. Safety and Efficacy

16.2.6. Product Profile

16.3. Rilutek (Riluzole): Sanofi/Covis

16.3.1. Drug Description

16.3.2. Regulatory Milestones

16.3.3. Other Developmental activities

16.3.4. Safety and Efficacy

16.3.5. Product Profile

16.4. Exservan (Riluzole oral film): Aquestive Therapeutics

16.4.1. Drug Description

16.4.2. Regulatory Milestones

16.4.3. Other Developmental Activities

16.4.4. Safety and Efficacy

16.4.5. Product Profile

16.5. Nuedexta (dextromethorphan hydrobromide/quinidine sulfate): Avanir Pharmaceuticals (a subsidiary of Otsuka America)

16.5.1. Drug Description

16.5.2. Regulatory Milestones

16.5.3. Other Developmental Activities

16.5.4. Safety and Efficacy

16.5.5. Product Profile

16.6. Tiglutik (riluzole): ITF Pharma (a US subsidiary Italfarmaco)

16.6.1. Drug Description

16.6.2. Regulatory Milestones

16.6.3. Other Developmental Activities

16.6.4. Safety and Efficacy

16.6.5. Product Profile

17. Emerging Drugs

17.1. Key Competitors

17.2. Tofersen (BIIB067): Biogen/Ionis

17.2.1. Product Description

17.2.2. Other Developmental Activities

17.2.3. Clinical Development

17.2.4.  Safety and Efficacy

17.2.5. Product Profile

17.3. ION363: Ionis Pharmaceuticals

17.3.1. Product Description

17.3.2. Clinical Development

17.3.3. Product Profile

17.4. Ibudilast (MN-166): MediciNova

17.4.1. Product Description

17.4.2. Other Developmental Activities

17.4.3. Clinical Development

17.4.4. Safety and Efficacy

17.4.5. Product Profile

17.5. Masitinib: AB Science

17.5.1. Product Description

17.5.2. Other Developmental Activities

17.5.3. Clinical Development

17.5.4. Safety and Efficacy

17.5.5. Product Profile

17.6. NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics

17.6.1. Product Description

17.6.2. Other Developmental Activities

17.6.3. Clinical Development

17.6.4. Safety and Efficacy

17.6.5. Product Profile

17.7. AMX0035: Amylyx Pharmaceuticals

17.7.1. Product Description

17.7.2. Other Developmental Activities

17.7.3. Clinical Development

17.7.4. Safety and Efficacy

17.7.5. Product Profile

17.8. EPI-589: DS Pharma/PTC Therapeutics

17.8.1. Product Description

17.8.2. Other Developmental Activities

17.8.3. Clinical Development

17.8.4. Safety and Efficacy

17.8.5. Product Profile

17.9. SLS-005/Trehalose: Seelos Therapeutics

17.9.1. Product Description

17.9.2. Other Developmental Activities

17.9.3. Clinical Development

17.9.4. Product Profile

17.10. Pridopidine: Prilenia Therapeutics

17.10.1. Product Description

17.10.2. Other Developmental Activities

17.10.3. Clinical Development

17.10.4. Safety and Efficacy

17.10.5. Product Profile

17.11. Verdiperstat: Biohaven Pharmaceuticals

17.11.1. Product Description

17.11.2. Other Developmental Activities

17.11.3. Clinical Development

17.11.4. Product Profile

17.12. Zilucoplan: UCB Pharma/Ra Pharmaceuticals

17.12.1. Product Description

17.12.2. Other Development Activities

17.12.3. Clinical Development

17.12.4. Product Profile

17.13. Gold Nanocrystals/CNM-Au8: Clene Nanomedicine Biosciences

17.13.1. Product Description

17.13.2. Other development activity

17.13.3. Clinical Development

17.13.4. Safety and Efficacy

17.13.5. Product Profile

17.14. ANX005: Annexon

17.14.1. Product Description

17.14.2. Other Development activity

17.14.3. Clinical Development

17.14.4. Safety and Efficacy

17.14.5. Product Profile

17.15. AT-1501: Eledon Pharmaceuticals

17.15.1. Product Description

17.15.2. Other Development Activities

17.15.3. Clinical Development

17.15.4. Safety and Efficacy

17.15.5. Product Profile

17.16. AP-101: AL-S Pharma

17.16.1. Product Description

17.16.2. Other Development Activities

17.16.3. Clinical Development

17.16.4. Safety and Efficacy

17.16.5. Product Profile

17.17. Pegcetacoplan (APL-2): Apellis Pharmaceuticals

17.17.1. Product Description

17.17.2. Clinical Development

17.17.3. Product Profile

17.18. Reldesemtiv: Cytokinetics/Astellas Pharma

17.18.1. Product Description

17.18.2. Other Developmental Activities

17.18.3. Clinical Development

17.18.4. Safety and Efficacy

17.18.5. Product Profile

17.19. RNS60: Revalesio Corporation

17.19.1. Product Description

17.19.2. Other Developmental Activities

17.19.3. Clinical Development

17.19.4. Safety and Efficacy

17.19.5. Product Profile

17.20. BIIB078: Biogen

17.20.1. Product Description

17.20.2. Other Developmental Activities

17.20.3. Clinical Development

17.20.4. Product Profile

17.21. PrimeC: NeuroSense Therapeutics

17.21.1. Product Description

17.21.2. Other Developmental Activities

17.21.3. Clinical Development

17.21.4. Safety and Efficacy

17.21.5. Product Profile

18. Amyotrophic Lateral Sclerosis (ALS): 7MM Market Analysis

18.1. Key Findings

18.2. Market Methodology

18.3. Total Market Size of Amyotrophic Lateral Sclerosis in the 7MM

18.4. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in the 7MM

18.5. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in the 7MM

18.6. Market Outlook

18.7. Attribute Analysis

18.8. Key Market Forecast Assumptions

18.9. United States Market Size

18.9.1. Total Market Size of Amyotrophic Lateral Sclerosis in the United States

18.9.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in the United States

18.9.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in the United States

18.10. EU-5 Market Size

18.10.1. Total Market size of Amyotrophic Lateral Sclerosis in Europe

18.10.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in Europe

18.10.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in Europe

18.11. Japan

18.11.1. Total Market size of Amyotrophic Lateral Sclerosis in Japan

18.11.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in Japan

18.11.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in Japan

19. Market Drivers

20. Market Barriers

21. Unmet Needs

22. Market Access and Reimbursement

23. Appendix

23.1. Bibliography

23.2. Report Methodology

24. DelveInsight Capabilities

25. Disclaimer

26. About DelveInsight

List of Table

Table 1: Summary of Amyotrophic lateral sclerosis (ALS) Market, and Epidemiology (2019-2032)

Table 2: Key Events

Table 3: List of differential diagnosis and clinical overlap with ALS

Table 4: Summary table of ALS and key differentiating factors with each mimic/variant

Table 5: Diagnostic tests for ALS

Table 6: Revised El Escorial Criteria for ALS

Table 7: Neuroprotective and Symptomatic Treatments for ALS

Table 8: Clinical practice and treatment guidelines for ALS in Japan, the United States, and Europe.

Table 9: Total Prevalent Population of ALS in the 7MM (2019-2032)

Table 10: Total Diagnosed Prevalent Population of ALS in the 7MM (2019-2032)

Table 11: Prevalence of ALS in the US (2019-2032)

Table 12: Diagnosed Prevalence of ALS in the US (2019-2032)

Table 13: Gender-specific Distribution of ALS in the US (2019-2032)

Table 14: Mutation-specific Distribution of ALS in the US (2019-2032)

Table 15: Type-specific Distribution of ALS in the US (2019-2032)

Table 16: Site of onset of ALS in the US (2019-2032)

Table 17: Age-specific Distribution of ALS in the US (2019-2032)

Table 18: Prevalence of ALS in EU5 (2019-2032)

Table 19: Diagnosed Prevalence of ALS in EU5 (2019-2032)

Table 20: Gender-specific Distribution of ALS in EU5 (2019-2032)

Table 21: Mutation-specific Distribution of ALS in EU5 (2019-2032)

Table 22: Type-specific Distribution of ALS in EU5 (2019-2032)

Table 23: Site of onset of ALS in EU5 (2019-2032)

Table 24: Age-specific Distribution of ALS in EU5 (2019-2032)

Table 25: Prevalence of ALS in Japan (2019-2032)

Table 26: Diagnosed Prevalence of ALS in Japan (2019-2032)

Table 27: Gender-specific Distribution of ALS in Japan (2019-2032)

Table 28: Mutation-specific Distribution of ALS in Japan (2019-2032)

Table 29: Type-specific Distribution of ALS in Japan (2019-2032)

Table 30: Site of onset of ALS in Japan (2019-2032)

Table 31: Age-specific Distribution of ALS in Japan (2019-2032)

Table 32: Organizations contributing toward ALS

Table 33: Comparison of marketed drugs

Table 34: Patent Expiration of Radicava

Table 35: Radicava (MT-1186), Clinical Trial Description, 2022

Table 36: Patent Expiration of Exservan

Table 37: Patent Expiration of Nuedexta

Table 38: Patent Expiration of Tiglutik

Table 39: Comparison of emerging drugs under development

Table 40: Tofersen, Clinical Trial Description, 2022

Table 41: ION363, Clinical Trial Description, 2022

Table 42: Ibudilast Clinical Trial Description, 2022

Table 43: Masitinib, Clinical Trial Description, 2022

Table 44: NurOwn, Clinical Trial Description, 2022

Table 45: AMX0035, Clinical Trial Description, 2022

Table 46: EPI-589, Clinical Trial Description, 2022

Table 47: SLS-005, Clinical Trial Description, 2022

Table 48: Pridopidine, Clinical Trial Description, 2022

Table 49: Verdiperstat, Clinical Trial Description, 2022

Table 50: Zilucoplan, Clinical Trial Description, 2022

Table 51: CNM-Au8, Clinical Trial Description, 2022

Table 52: CNM-Au8, Clinical Trial Description, 2022

Table 53: AT-1501, Clinical Trial Description, 2022

Table 54: AP-101, Clinical Trial Description, 2022

Table 55: Pegcetacoplan, Clinical Trial Description, 2022

Table 56: Reldesemtiv, Clinical Trial Description, 2022

Table 57: RNS60, Clinical Trial Description, 2022

Table 58: BIIB078, Clinical Trial Description, 2022

Table 59: PrimeC, Clinical Trial Description, 2022

Table 60: Market Size of ALS in the 7MM in USD Million (2019-2032)

Table 61: Market Size of ALS by Current Therapies in the 7MM, in USD Million (2019-2032)

Table 62: Market Size of ALS by Emerging Therapies in the 7MM, in USD Million (2019-2032)

Table 63: Attribute analysis of Emerging drugs

Table 64: Key Market Forecast Assumptions for Tofersen/BIIB067

Table 65: Key Market Forecast Assumptions for Ibudilast

Table 66: Key Market Forecast Assumptions for Masitinib

Table 67: Key Market Forecast Assumptions for NurOwn

Table 68: Key Market Forecast Assumptions for AMX0035

Table 69: Key Market Forecast Assumptions for EPI-589

Table 70: Key Market Forecast Assumptions for ANX005

Table 71: Key Market Forecast Assumptions for AT-1501

Table 72: Key Market Forecast Assumptions for AP-101

Table 73: Key Market Forecast Assumptions for BIIB078

Table 74: Key Market Forecast Assumptions for ION363

Table 75: Key Market Forecast Assumptions for PrimeC

Table 76: Key Market Forecast Assumptions for Pegcetacoplan

Table 77: Key Market Forecast Assumptions for Zilucoplan

Table 78: Key Market Forecast Assumptions for CNM-Au8

Table 79: Key Market Forecast Assumptions for Pridopidine

Table 80: Key Market Forecast Assumptions for SLS-005

Table 81: Key Market Forecast Assumptions for Verdiperstat

Table 82: Key Market Forecast Assumptions for RNS60

Table 83: Key Market Forecast Assumptions for Reldesemtiv

Table 84: Market Size of ALS in the US, USD Million (2019-2032)

Table 85: Market Size of ALS by Current Therapies in the US, in USD Million (2019-2032)

Table 86: Market Size of ALS by Emerging Therapies in the US, in USD Million (2019-2032)

Table 87: EU5 Market Size of ALS in USD Million (2019-2032)

Table 88: EU5 Market Size of ALS by Current Therapies in USD Million (2019-2032)

Table 89: EU5 Market Size of ALS by Emerging Therapies in USD Million (2019-2032)

Table 90: Market Size of ALS by Current Therapies in Japan, in USD Million (2019-2032)

Table 91: Market Size of ALS by Emerging Therapies in the US, in USD Million (2019-2032)

Table 92: Market Size of ALS by Emerging Therapies in the US, in USD Million (2019-2032)

List of Figures

Figure 1: Phenotypic presentations of ALS.

Figure 2: Clustering of ALS genes in pathogenic pathways.

Figure 3: Flowchart for the management of respiratory dysfunction in ALS/ motor neuron disease (MND)

Figure 4: Total Prevalent Population of ALS in the 7MM (2019-2032)

Figure 5: Total Diagnosed Prevalent Population of ALS in the 7MM (2019-2032)

Figure 6: Prevalence of ALS in the US (2019-2032)

Figure 7: Diagnosed Prevalence of ALS in the US (2019-2032)

Figure 8: Gender-specific Distribution of ALS in the US (2019-2032)

Figure 9: Mutation-specific Distribution of ALS in the US (2019-2032)

Figure 10: Type-specific Distribution of ALS in the US (2019-2032)

Figure 11: Site of onset of ALS in the US (2019-2032)

Figure 12: Age-specific Distribution of ALS in the US (2019-2032)

Figure 13: Prevalence of ALS in EU5 (2019-2032)

Figure 14: Diagnosed Prevalence of ALS in EU5 (2019-2032)

Figure 15: Gender-specific Distribution of ALS in EU5 (2019-2032)

Figure 16: Mutation-specific Distribution of ALS in EU5 (2019-2032)

Figure 17: Type-specific Distribution of ALS in EU5 (2019-2032)

Figure 18: Site of onset of ALS in EU5 (2019-2032)

Figure 19: Age-specific Distribution of ALS in EU5 (2019-2032)

Figure 20: Prevalence of ALS in Japan (2019-2032)

Figure 21: Diagnosed Prevalence of ALS in Japan (2019-2032)

Figure 22: Gender-specific Distribution of ALS in Japan (2019-2032)

Figure 23: Mutation-specific Distribution of ALS in Japan (2019-2032)

Figure 24: Type-specific Distribution of ALS in Japan (2019-2032)

Figure 25: Site of onset of ALS in Japan (2019-2032)

Figure 26: Age-specific Distribution of ALS in Japan (2019-2032)

Figure 27: Market Size of ALS in the 7MM in USD Million (2019-2032)

Figure 28: Market Size of ALS by Current Therapies in the 7MM, in USD Million (2019-2032)

Figure 29: Market Size of ALS by Emerging Therapies in the 7MM, in USD Million (2019-2032)

Figure 30: Market Size of ALS in the US, USD Millions (2019-2032)

Figure 31: Market Size of ALS by Current Therapies in the US, USD Million (2019-2032)

Figure 32: Market Size of ALS by Emerging Therapies in the US, USD Million (2019-2032)

Figure 33: Market Size of ALS in EU5, USD Million (2019-2032)

Figure 34: EU5 Market Size of ALS by Current Therapies in USD Million (2019-2032)

Figure 35: EU5 Market Size of ALS by Emerging Therapies in USD Million (2019-2032)

Figure 36: Market Size of ALS in Japan, USD Million (2019-2032)

Figure 37: Japan Market Size of ALS by Current Therapies, USD Million (2019-2032)

Figure 38: Japan Market Size of ALS by Emerging Therapies, USD Million (2019-2032)

Mitsubishi Tanabe Pharma Corporation
 Sanofi/Covis
 Aquestive Therapeutics
 Avanir Pharmaceuticals (a subsidiary of Otsuka America)
 Biogen
 Ionis Pharmaceuticals
 MediciNova
 AB Science
 Brainstorm Cell Therapeutics
 Amylyx Pharmaceuticals
 DS Pharma/PTC Therapeutics
 Seelos Therapeutics
 Prilenia Therapeutics
 Biohaven Pharmaceuticals
 UCB Pharma/Ra Pharmaceuticals
 Clene Nanomedicine Biosciences
 Annexon
 Eledon Pharmaceuticals
 AL-S Pharma
 Apellis Pharmaceuticals
 Cytokinetics/Astellas Pharma
 Revalesio Corporation
 NeuroSense Therapeutics

 

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