筋萎縮性側索硬化症（ALS）市場 - 市場の洞察、疫学、市場予測：2034年

筋萎縮性側索硬化症（ALS）はルー・ゲーリッグ病とも呼ばれ、脳と脊髄の神経細胞に影響を及ぼす進行性の神経変性疾患であり、筋力低下、麻痺、最終的には呼吸不全に至る。正確な原因はいまだ不明ですが、遺伝的要因と環境的要因の組み合わせが発症に関与している可能性が高いです。診断には神経学的検査と他の疾患を除外するための様々な検査が必要です。現在、ALSを治療する方法は確立されていませんが、治療は症状の管理と生活の質の向上に重点を置いています。可能性のある治療法を模索し、ALSをより深く理解するための調査が続けられています。

ALSの診断には、徹底的な神経学的検査、病歴の確認、他の疾患の可能性を除外するための様々な検査が必要です。筋電図検査（EMG）と神経伝導検査は神経と筋肉の機能を評価し、MRIなどの画像検査は症状の他の原因を除外します。このような努力にもかかわらず、決定的な検査がない限り、ALSの診断は依然として困難です。早期かつ正確な診断は、症状管理や支援サービスを開始する上で極めて重要です。脳脊髄液ニューロフィラメント値のようなバイオマーカーは、まだ臨床に組み込まれてはいないが、診断や治療効果のモニタリングに有望です。特に症状が曖昧であったり、他の疾患と重複していたりする場合には、患者の層別化や調査研究に役立つ可能性があります。

主要7ヶ国におけるALSの市場規模は、2023年のに10億米ドルとなっています。リルゾールの処方を含む現在の標準治療は、病気の進行を遅らせることで患者の寿命を延ばすことに有効です。

現在、ALSの治療薬として使用されているのは、EXSERVAN（リルゾール経口フィルム）、RELYVRIO、QALSODY、RADICAVA（エダラボン注射剤）、RADICAVA ORS（エダラボン経口懸濁剤）、NEUDEXTA、TIGLUTEK、RILUTEKのほか、抗てんかん薬、オピオイド、NSAIDs、利尿薬、SSRI、抗うつ薬などの対症療法です。

当レポートでは、主要7ヶ国における筋萎縮性側索硬化症（ALS）市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2034年までの市場規模予測、および医療のアンメットニーズなどを提供しています。

目次

第1章 重要な洞察

第2章 報告書のイントロダクション

第3章 ALS市場概要

第4章 疫学と市場予測の調査手法

第5章 エグゼクティブサマリー

第6章 主な出来事

第7章 疾患の背景と概要

• イントロダクション
• ALSの種類
• 原因
• リスク要因
• 症状
• 臨床的特徴
• 病因
• バイオマーカー
• 予後の予測
• 鑑別診断
• 診断

第8章 ALSの治療と管理

• 多分野にわたるケア
• 神経保護治療
• アンチセンスオリゴヌクレオチド
• 対症療法
• 補助器具
• 栄養
• 呼吸器管理
• 秘密管理
• 緩和ケアと終末期ケア
• ガイドライン

第9章 主要7ヶ国の疫学と患者人口

• 主な調査結果
• 仮定と根拠
• 主要7ヶ国におけるALSの有病者総数
• 主要7ヶ国におけるALSの診断を受けた有病者総数
• 米国
• EU4ヶ国と英国
• 日本

第10章 患者動向

第11章 ALSにおける主要なエンドポイント

第12章 上市済み薬剤

第13章 新興薬剤

第14章 ALS：主要7ヶ国分析

• 主な調査結果
• 市場見通し
• コンジョイント分析
• 主要な市場予測の前提条件
• 主要7ヶ国におけるALSの総市場規模
• 米国の市場規模
• EU4ヶ国と英国の市場規模
• 日本の市場規模

第15章 アンメットニーズ

第16章 SWOT分析

第17章 KOLの見解

第18章 市場アクセスと償還

第19章 付録

第20章 DELVEINSIGHTの機能

第21章 免責事項

第22章 DELVEINSIGHTについて

List of Tables

• Table 1: Summary of Amyotrophic lateral sclerosis (ALS) Market, and Epidemiology (2020-2034)
• Table 2: List of differential diagnosis and clinical overlap with ALS
• Table 3: Summary table of ALS and key differentiating factors with each mimic/variant
• Table 4: Diagnostic tests for ALS
• Table 5: Revised El Escorial Criteria for ALS
• Table 6: Neuroprotective and Symptomatic Treatments for ALS
• Table 7: Clinical practice and treatment guidelines for ALS in Japan, the United States, and Europe.
• Table 8: Total Prevalent Population of ALS in the 7MM (2020-2034)
• Table 9: Total Diagnosed Prevalent Population of ALS in the 7MM (2020-2034)
• Table 10: Total Diagnosed Prevalent Population of ALS in the United States (2020-2034)
• Table 11: Type-specific Distribution of ALS in the United States (2020-2034)
• Table 12: Gender-specific Distribution of ALS in the United States (2020-2034)
• Table 13: Mutation-specific Distribution of ALS in the United States (2020-2034)
• Table 14: Distribution Based on Site of Onset of ALS in the United States (2020-2034)
• Table 15: Age-specific Distribution of ALS in the United States (2020-2034)
• Table 16: Diagnosed Prevalence of ALS in EU4 and the UK (2020-2034)
• Table 17: Type-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Table 18: Gender-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Table 19: Mutation-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Table 20: Distribution Based on Site of Onset of ALS in EU4 and the UK (2020-2034)
• Table 21: Mutation-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Table 22: Total Diagnosed Prevalent Population of ALS in Japan (2020-2034)
• Table 23: Type-specific Distribution of ALS in Japan (2020-2034)
• Table 24: Gender-specific Distribution of ALS in Japan (2020-2034)
• Table 25: Mutation-specific Distribution of ALS in Japan (2020-2034)
• Table 26: Distribution Based on Site of Onset of ALS in the Japan (2020-2034)
• Table 27: Age-specific Distribution of ALS in Japan (2020-2034)
• Table 28: Comparison of Marketed Drugs in the 7MM
• Table 29: Patent Expiration of QALSODY
• Table 30: QALSODY, Clinical Trial Description, 2024
• Table 31: RELYVRIO, Clinical Trial Description, 2024
• Table 32: Patent Expiration of EXSERVAN
• Table 33: Patent Expiration of NUEDEXTA
• Table 34: Patent Expiration of TIGLUTIK
• Table 35: Patent Expiration of RADICAVA and EDARAVONE (RADICAVA ORS)
• Table 36: Comparison of Emerging Drugs Under Development for ALS
• Table 37: Masitinib (AB1010), Clinical Trial Description, 2024
• Table 38: Latozinemab (AL001/GSK4527223), Clinical Trial Description, 2024
• Table 39: NurOwn, Clinical Trial Description, 2024
• Table 40: ION363, Clinical Trial Description, 2024
• Table 41: MN-166 (ibudilast), Clinical Trial Description, 2024
• Table 42: DNL343, Clinical Trial Description, 2024
• Table 43: ABBV-CLS-7262, Clinical Trial Description, 2024
• Table 44: CNM-Au8, Clinical Trial Description, 2024
• Table 45: SLS-005 (trehalose), Clinical Trial Description, 2024
• Table 46: Pridopidine, Clinical Trial Description, 2024
• Table 47: RAPA-501, Clinical Trial Description, 2024
• Table 48: PrimeC, Clinical Trial Description, 2024
• Table 49: ENGENSIS (VM202), Clinical Trial Description, 2024
• Table 50: TPN-101, Clinical Trial Description, 2024
• Table 51: RNS60, Clinical Trial Description, 2024
• Table 52: ANX005, Clinical Trial Description, 2024
• Table 53: Dazucorilant, Clinical Trial Description, 2024
• Table 54: AP-101, Clinical Trial Description, 2024
• Table 55: SAR443820/DNL788, Clinical Trial Description, 2024
• Table 56: AIT-101, Clinical Trial Description, 2024
• Table 57: Key Market Forecast Assumption of ALS in the United States
• Table 58: Key Market Forecast Assumption of ALS in EU4 and the UK
• Table 59: Key Market Forecast Assumption of ALS in Japan
• Table 60: Total Market Size of ALS in the 7MM, USD million (2020-2034)
• Table 61: Total Market Size of ALS in the United States, USD million (2020-2034)
• Table 62: Market Size of ALS by Therapies in the United States, USD million (2020-2034)
• Table 63: Total Market Size of ALS in EU4 and the UK, USD million (2020-2034)
• Table 64: Market Size of ALS by Therapies in EU4 and the UK, USD million (2020-2034)
• Table 65: Total Market Size of ALS in Japan, USD million (2020-2034)
• Table 66: Market Size of ALS by Therapies in Japan, USD million (2020-2034)

List of Figures

• Figure 1: Phenotypic presentations of ALS
• Figure 2: Clustering of ALS genes in pathogenic pathways
• Figure 3: Total Prevalent Population of ALS in the 7MM (2020-2034)
• Figure 4: Total Diagnosed Prevalent Population of ALS in the 7MM (2020-2034)
• Figure 5: Diagnosed Prevalence of ALS in the United States (2020-2034)
• Figure 6: Type-specific Distribution of ALS in the United States (2020-2034)
• Figure 7: Gender-specific Distribution of ALS in the United States (2020-2034)
• Figure 8: Mutation-specific Distribution of ALS in the United States (2020-2034)
• Figure 9: Distribution Based on Site of Onset of ALS in the United States (2020-2034)
• Figure 10: Age-specific Distribution of ALS in the United States (2020-2034)
• Figure 11: Diagnosed Prevalence of ALS in EU4 and the UK (2020-2034)
• Figure 12: Type-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Figure 13: Gender-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Figure 14: Mutation-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Figure 15: Distribution Based on Site of Onset of ALS in EU4 and the UK (2020-2034)
• Figure 16: Age-specific Distribution of ALS in EU4 and the UK (2020-2034)
• Figure 17: Diagnosed Prevalence of ALS in Japan (2020-2034)
• Figure 18: Type-specific Distribution of ALS in Japan (2020-2034)
• Figure 19: Gender-specific Distribution of ALS in Japan (2020-2034)
• Figure 20: Mutation-specific Distribution of ALS in Japan (2020-2034)
• Figure 21: Distribution Based on Site of Onset of ALS in Japan (2020-2034)
• Figure 22: Age-specific Distribution of ALS in Japan (2020-2034)
• Figure 23: Total Market Size of ALS in the 7MM (2020-2034)
• Figure 24: Total Market Size of ALS in the United States (2020-2034)
• Figure 25: Total Market Size of ALS in the United States (2020-2034)
• Figure 26: Total Market Size of ALS in EU4 and the UK (2020-2034)
• Figure 27: Total Market Size of ALS in EU4 and the UK (2020-2034)
• Figure 28: Total Market Size of ALS in Japan (2020-2034)
• Figure 29: Total Market Size of ALS in Japan (2020-2034)

Key Highlights:

• The market size of ALS in 2023, in the 7MM was ALS ~ USD 1,000 million.
• The current standard of care that involves the prescription of riluzole has been effective in slowing the disease progression, thereby increasing the life span of patients.
• The most recent development in ALS was the addition of RELYVRIO for the treatment of ALS. On September 29, 2022, the US FDA approved RELYVRIO (sodium phenylbutyrate and taurursodiol) for the treatment of adults with ALS.
• After the setback with RELYVRIO, RADICAVA emerges as the leading contender in the current ALS market and is anticipated to achieve the highest revenue.
• After the approval of RADICAVA ORS, there was a notable increase in sales for RADICAVA, which had previously been declining for IV RADICAVA. The launch of RADICAVA ORS resulted in a high uptake of the drug in both the US and Japan
• Transposon Therapeutics' TPN-101 and Ionis Pharmaceuticals' Ulefnersen (ION363) are pioneering therapies tailored for specific mutations like C9ORF72 and FUS in ALS. These developments highlight a significant unmet need among patients with mutation-specific ALS, with current attention primarily focused on key mutations such as SOD1, C9ORF72, and FUS. However, there remains a notable gap in targeting mutations like TARDBP, OPTN, ANG, and others, presenting a compelling opportunity for future research and therapeutic advancements in the ALS landscape. Whereas in the majority of ALS patients, mutations still remain undiscovered.
• Currently, the drugs used for the treatment of ALS include EXSERVAN (Riluzole oral film), RELYVRIO, QALSODY, RADICAVA (edaravone injection), and RADICAVA ORS (edaravone oral suspension), NEUDEXTA, TIGLUTEK, RILUTEK along with other symptomatic treatment such as anti-epileptic drugs, opioids, NSAIDs, diuretics, SSRIs, antidepressants, etc. for the treatment of ALS.
• Expected launch of potential therapies, Ulefnersen (Ionis Pharmaceuticals), Ibudilast (MediciNova), Masitinib (AB Science), NurOwn (Brainstorm-Cell Therapeutics), CNM-Au8 (Clene Nanomedicine), and others may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of ALS. It is expected that these therapies will help the market of ALS post-launch, in the 7MM during the forecast period (2024-2034).

DelveInsight's "Amyotrophic Lateral Sclerosis - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of ALS, historical and forecasted epidemiology as well as the ALS market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The ALS market report provides current treatment practices, emerging drugs, ALS market share of individual therapies, and current and forecasted ALS market size from 2020 to 2034, segmented by seven major markets. The report also covers current ALS treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Amyotrophic Lateral Sclerosis (ALS) Understanding and Treatment Algorithm

Amyotrophic Lateral Sclerosis Overview

ALS, often referred to as Lou Gehrig's disease, is a progressive neurodegenerative disorder affecting the nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and ultimately respiratory failure. While the exact cause remains unclear, a combination of genetic and environmental factors likely contributes to its onset. Diagnosis relies on neurological examination and various tests to rule out other conditions. Although there is currently no cure for ALS, treatment focuses on managing symptoms and improving quality of life. Research efforts continue to explore potential therapies and better understand the disease.

Amyotrophic Lateral Sclerosis Diagnosis

Diagnosing ALS involves a thorough neurological examination, medical history review, and various tests to exclude other possible conditions. Electromyography (EMG) and nerve conduction studies evaluate nerve and muscle function, while imaging scans like MRI rule out alternative causes of symptoms. Despite these efforts, ALS diagnosis remains challenging without a definitive test. Early and accurate diagnosis is crucial for initiating symptom management and support services. Biomarkers, like cerebrospinal fluid neurofilament levels, though not yet integrated into clinical practice, show promise for diagnosis and monitoring treatment effects. They could aid in patient stratification and research studies, particularly in cases where symptoms are ambiguous or overlap with other conditions.

Further details related to diagnosis will be provided in the report...

Amyotrophic Lateral Sclerosis Treatment

Treatment for ALS aims to manage symptoms, improve quality of life, and slow disease progression. Medications such as riluzole and edaravone may be prescribed to help delay progression, although their effects are modest. Physical therapy, occupational therapy, and speech therapy can help maintain mobility, function, and communication abilities. Assistive devices such as wheelchairs, braces, and communication aids are often utilized to enhance independence. Additionally, respiratory support may be necessary as the disease progresses. While there is currently no cure for ALS, a multidisciplinary approach involving healthcare professionals, caregivers, and support networks is essential in providing comprehensive care and support to individuals living with the disease.

Further details related to treatment will be provided in the report.....

Amyotrophic Lateral Sclerosis Epidemiology

The ALS epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the Total Prevalent Population of ALS, Diagnosed Prevalence of ALS, Type-specific Distribution of ALS, Gender-specific Distribution of ALS, Mutation-specific Distribution of ALS, Distribution Based on Site of Onset of ALS, and Age-specific Distribution of ALS in the 7MM market covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Among the 7MM, the US accounted for the highest prevalent cases of ALS in 2023, with around 26,000 cases; these cases are expected to increase during the forecast period.
• Amongst EU4 and the UK, the diagnosed prevalent cases of ALS were highest in Germany, while the lowest number of cases was in Spain in 2023.
• The major sites of onset of ALS are the bulbar, and spinal, along with other uncertain regions. In 2023, the patients with the spinal as a site of onset accounted highest cases, i.e., 15,000, followed by the bulbar site of onset.
• According to the estimates, in Japan, it is observed that ALS was most prevalent in the 70-79 years age group, followed by 60-69 years, and 50-59 years.

Amyotrophic Lateral Sclerosis Drug Chapters

The drug chapter segment of the ALS report encloses a detailed analysis of the marketed and late-stage (Phase III) pipeline drug. The marketed drugs segment encloses drugs such as RADICAVA (Mitsubishi Tanabe Pharma Corporation), RELYVRIO (Amylyx Pharmaceuticals), TIGLUTIK (ITF Pharma), and others. Furthermore, the current key players for emerging drugs and their respective drug candidates include AB Science (Masitinib), Brainstorm Cell Therapeutics (NurOwn), and others. The drug chapter also helps understand the ALS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, and the latest news and press releases.

Marketed Drugs

RADICAVA/RADICUT (edaravone injection) and RADICAVA ORS: Mitsubishi Tanabe Pharma Corporation

RADICAVA is a product of Mitsubishi Tanabe Pharma that contains the active substance edaravone. It is a novel neuroprotective agent that slows down ALS progression. Notably, RADICAVA is the second drug to be approved for the treatment of ALS after more than two decades from the first US FDA approval of riluzole. Edaravone is available as a clear, colorless liquid provided as a sterile injection solution supplied for IV infusion; the substituted 2-pyrazolin-5-one class has the chemical name 3-methyl-1-phenyl-2-pyrazolin-5-one. The mechanism by which RADICAVA exerts its therapeutic effect in patients with ALS is unknown; however, it has anti-oxidant properties (Cruz, 2018b). Even after getting approval, the company is still conducting various clinical trial studies to better understand the drug's effects on a larger scale.

RADICAVA received marketing approval in May 2017 and an exclusivity end date is in May 2024. RADICAVA ORS received marketing approval in May 2022.

RELYVRIO: Amylyx Pharmaceuticals

RELYVRIO (AMX0035) is an investigational neuroprotective therapy being developed to minimize neuronal death and dysfunction. In ALS and other neurodegenerative disorders, the drug targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways and blocks stress to maintain a balance between them. AMX0035 is a fixed-dose co formulation of two active compounds, namely, sodium phenylbutyrate (PB) and taurursodiol (tauroursodeoxycholic acid [TUDCA].

Currently, the drug is being studied in a Phase III PHOENIX study for treating ALS. Recently, in March 2024, the company reported topline results from the Phoenix trial, where the study did not meet prespecified primary or secondary endpoints.

Within the next 8 weeks, Amylyx will continue to engage with regulatory authorities and the ALS community to share topline data. The company will share plans for RELYVRIO in ALS, which may include voluntarily withdrawing RELYVRIO from the market. At this time, RELYVRIO will continue to be available for people living with ALS. The company has voluntarily decided to pause promotion; however, related patient support services will remain in place.

Emerging Drugs

Masitinib: AB Science

Masitinib (AB1010) is an orally administered tyrosine kinase inhibitor. It modulates mast cells and macrophages' activity - important cells for immunity - by targeting a limited number of kinases without inhibiting, at therapeutic doses, kinases associated with known toxicities. Masitinib distinguishes itself from other ALS developmental drugs by exerting neuroprotection in both central and peripheral nervous systems. Based on its unique mechanism of action, masitinib can be developed in many conditions in oncology, inflammatory diseases, and certain diseases of the central nervous system. AB Science completed a Phase II/III trial and has attained positive results, followed by a green signal from the US FDA on the IND application. However, the drug is under investigation for a Phase III trial in patients with ALS.

In January 2024, AB Science SA announced that the Committee for Medicinal Products for Human Use (CHMP) has proposed that AB Science submit a written response to the List of Outstanding Issues at D195 of the procedure instead of addressing these issues through the oral explanation. AB Science expects an opinion from the CHMP in the second quarter of 2024.

NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics

MSC-NTF cells (NurOwn) are autologous bone marrow-derived mesenchymal stem cells (MSC) induced in culture to secrete high levels of neurotrophic factors (NTFs) that support neuronal growth and survival. Thus, MSC-NTF cells combine MSC's immunomodulatory therapeutic benefits with enhanced neurotrophic factor secretion.

In October 2023, the company announced a strategic realignment to enable accelerated development of NurOwn for the treatment of ALS. This realignment is designed to

1) Support the company's plans to conduct a double-blind, placebo-controlled Phase IIIb US clinical trial for NurOwn in ALS with an open-label extension.

2) Continue to publish data from NurOwn's Phase III clinical trial on biomarkers, long-term safety and survival, and the expanded access program, providing transparency around NurOwn data and progressing ALS drug development.

Drug Class Insight

Tyrosine kinase inhibitor

Tyrosine kinase inhibitors (TKIs) have been investigated in ALS research as potential therapeutic agents. TKIs target specific enzymes involved in cell signaling pathways, which may play a role in neurodegeneration seen in ALS. One TKI that has been studied in ALS is masitinib. Masitinib is a TKI that inhibits several tyrosine kinases, including c-Kit, platelet-derived growth factor receptor (PDGFR), and Lyn kinase. These kinases are involved in various cellular processes, including cell growth, survival, and inflammation, which are relevant to the pathophysiology of ALS. Clinical trials investigating masitinib in ALS have shown promising results in terms of slowing disease progression and improving survival rates in some patients. However, further research is needed to confirm its efficacy and safety in larger populations of ALS patients.

Amyotrophic Lateral Sclerosis Market Outlook

Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. Scarcely any drugs have been approved by the FDA that can slow the course of the disease and improve the quality of life. Therefore, the management of ALS remains supportive and symptom-based. In recent years, research on new treatment strategies has increased, taking heed of gene therapy, cellular therapy, and neuroprotective agents. There are limited approved drugs that slow disease progression by prolonging autonomy and increasing survival rates (measured by the ALS functional rating scale (ALSFRS-R). Moreover, approved by the US FDA to treat ALS, including riluzole, NUEDEXTA, RADICAVA, TIGLUTIK, RELYVRIO, and QALSODY. Medications are also prescribed to help manage symptoms of ALS, including pain, muscle cramps, stiffness, excess saliva and phlegm, and the pseudobulbar effect (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.

For the treatment of ALS, Riluzole is recommended as first-line therapy in all three regions - Japan, the US, and Europe. Riluzole was first approved in the US by the FDA in 1995, and it was later approved in many other countries in the ensuing decades. It is a medication that appears to prolong the life of some people with ALS by at least a few months. Riluzole seems to do two things: block sodium and calcium channels and increase glutamate clearance. Currently, no other drug 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.

Although the pipeline holds multiple promising therapies in various stages of development, the failure rates of clinical trials are quite high for ALS. Therapies like arimoclomol (Orphazyme), levosimendan (Orion Pharmaceuticals), ravulizumab (Ultomiris), and Zilucoplan (UCB Pharma), are some recent failures in the list of therapies for ALS, and more may follow in the future. Nevertheless, the current pipeline holds great potential as it contains disease-modifying agents, symptomatic treatments, and therapies targeting specific mutations, which could help in fulfilling the unmet treatment needs of ALS patients.

Detailed market assessment will be provided in the final report.

Key Findings

• The total market size in the US for ALS was estimated to be ~USD 920 million in 2023, which is expected to grow during the forecast period (2024-2034).
• RADICAVA emerges as the leading contender in the current ALS market and is anticipated to achieve the highest revenue, i.e., USD 923 million in the US by 2034.
• In 2034, among the emerging therapies, the highest revenue was generated by Masitinib in combination with riluzole, i.e., USD 37 million in Japan.

Amyotrophic Lateral Sclerosis Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034. The landscape of ALS treatment has experienced a transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of Neurologists, professors of neurology, and director of the Neuromuscular Reference Center at the University Hospital, and professors in the Department of Translational Neuroscience. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.

Amyotrophic Lateral Sclerosis Pipeline Development Activities

The report provides insights into therapeutic candidates in Phase III, Phase II, and Phase I/II. It also analyzes key players involved in developing targeted therapeutics. Companies like AB Science and Brainstorm Cell Therapeutics actively engage in late-stage research and development efforts for ALS. The pipeline of ALS possesses many potential drugs and there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for ALS emerging therapy.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the ALS evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Neurology specialists, Neuroscience specialists, and others.

DelveInsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as the University of Nebraska Medical Center, Centers for Disease Control and Prevention, Department of Translational Neuroscience, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or ALS market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Market Access and Reimbursement

The treatment and management of ALS are expensive. The significant expense for ALS treatment comes from the patient's pocket, with palliative care and management having a major share of the out-of-pocket expenditure. Expensive drugs like RADICAVA pose a burden for ALS patients as not everyone can get access to the drug due to its high cost.

The Patient Advocate Foundation's (PAF) Copay Relief (CPR) Program also has an ALS fund that provides copay, co-insurance, 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. The eligibility criteria for receiving the fund are that the patient must be insured, the insurance must cover the medication for ALS, and the patient must reside and receive treatment in the US. Currently, the fund is not accepting applications due to insufficient donations.

Medicare Part B coverage for RADICAVA

Under Medicare Part B, 80% of costs for a RADICAVA treatment are covered (after the annual deductible is paid), while the remaining 20% must be covered either by the patient or with supplemental coverage.

Detailed market access and reimbursement assessment will be provided in the final report.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of ALS, explaining its causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the ALS market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive ALS.

Amyotrophic Lateral Sclerosis Report Insights

• Patient Population
• Therapeutic Approaches
• Amyotrophic Lateral Sclerosis Pipeline Analysis
• Amyotrophic Lateral Sclerosis Market Size and Trends
• Existing and Future Market Opportunity

Amyotrophic Lateral Sclerosis Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Amyotrophic Lateral Sclerosis Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Amyotrophic Lateral Sclerosis Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Analyst Views)

FAQs

• What was the ALS market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for ALS?
• What are the disease risks, burdens, and unmet needs of ALS? What will be the growth opportunities across the 7MM concerning the patient population with ALS?
• What are the current options for the treatment of ALS? What are the current guidelines for treating ALS in the 7MM?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• What is the patient share in Amyotrophic Lateral Sclerosis?

Reasons to Buy:

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving ALS.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential current and emerging therapies under the analyst view section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1. KEY INSIGHTS

2. REPORT INTRODUCTION

3. ALS MARKET OVERVIEW AT A GLANCE

• 3.1. MARKET SHARE (%) DISTRIBUTION OF ALS IN 2020 BY THERAPIES
• 3.2. MARKET SHARE (%) DISTRIBUTION OF ALS IN 2034 BY THERAPIES

4. EPIDEMIOLOGY AND MARKET FORECAST METHODOLOGY

5. EXECUTIVE SUMMARY

6. KEY EVENTS

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
• 7.1. 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
• 8.3. ANTISENSE OLIGONUCLEOTIDES
• 8.4. SYMPTOMATIC TREATMENTS
• 8.5. ADAPTIVE AND ASSISTIVE EQUIPMENT
• 8.6. NUTRITION
• 8.7. RESPIRATORY MANAGEMENT
• 8.8. SECRETION MANAGEMENT
• 8.9. PALLIATIVE AND END-OF-LIFE CARE
• 8.1. GUIDELINES
  • 8.10.1. European Federation of Neurological Societies (EFNS) guidelines on the Clinical Management of ALS (MALS) (2012)
  • 8.10.2. American Association of Neurology (AAN) Guidelines for Care and Management of patients with ALS (2009)
  • 8.10.3. Management guidelines for ALS in Japan, the United States, and Europe.

9. EPIDEMIOLOGY AND PATIENT POPULATION OF THE 7MM

• 9.1. KEY FINDINGS
• 9.2. ASSUMPTIONS AND RATIONALE
• 9.3. TOTAL PREVALENT POPULATION OF ALS IN THE 7MM
• 9.4. TOTAL DIAGNOSED PREVALENT POPULATION OF ALS IN THE 7MM
• 9.5. UNITED STATES
  • 9.5.1. Diagnosed Prevalence of ALS in the United States
  • 9.5.2. Type-specific Distribution of ALS in the United States
  • 9.5.3. Gender-specific Distribution of ALS in the United States
  • 9.5.4. Mutation-specific Distribution of ALS in the United States
  • 9.5.5. Distribution Based on Site of Onset of ALS in the United States
  • 9.5.6. Age-specific Distribution of ALS in the United States
• 9.6. EU4 AND UK
  • 9.6.1. Diagnosed Prevalence of ALS in EU4 and the UK
  • 9.6.2. Type-specific Distribution of ALS in EU4 and the UK
  • 9.6.3. Gender-specific Distribution of ALS in EU4 and the UK
  • 9.6.4. Mutation-specific Distribution of ALS in EU4 and the UK
  • 9.6.5. Distribution Based on Site of Onset of ALS in EU4 and the UK
  • 9.6.6. Age-specific Distribution of ALS in EU4 and the UK
• 9.7. JAPAN
  • 9.7.1. Diagnosed Prevalence of ALS in Japan
  • 9.7.2. Type-specific Distribution of ALS in Japan
  • 9.7.3. Gender-specific Distribution of ALS in Japan
  • 9.7.4. Mutation-specific Distribution of ALS in Japan
  • 9.7.5. Distribution Based on Site of Onset of ALS in Japan
  • 9.7.6. Age-specific Distribution of ALS in Japan

10. PATIENT JOURNEY

11. KEY ENDPOINTS IN ALS

12. MARKETED DRUGS

• 12.1. KEY CROSS
• 12.2. QALSODY (TOFERSEN): BIOGEN/IONIS
  • 12.2.1. Drug Description
  • 12.2.2. Regulatory Milestones
  • 12.2.3. Other Developmental Activities
  • 12.2.4. Clinical Development
  • 12.2.5. Safety and Efficacy
  • 12.2.6. Product Profile
• 12.3. RELYVRIO: AMYLYX PHARMACEUTICALS
  • 12.3.1. Drug Description
  • 12.3.2. Regulatory Milestones
  • 12.3.3. Other Developmental Activities
  • 12.3.4. Clinical Development
  • 12.3.5. Safety and Efficacy
  • 12.3.6. Product Profile
• 12.4. EXSERVAN (RILUZOLE ORAL FILM): AQUESTIVE THERAPEUTICS/MITSUBISHI TANABE PHARMA/ZAMBON
  • 12.4.1. Drug Description
  • 12.4.2. Regulatory Milestones
  • 12.4.3. Other Developmental Activities
  • 12.4.4. Safety and Efficacy
  • 12.4.5. Product Profile
• 12.5. NUEDEXTA (DEXTROMETHORPHAN HYDROBROMIDE/QUINIDINE SULFATE): AVANIR PHARMACEUTICALS (A SUBSIDIARY OF OTSUKA AMERICA)
  • 12.5.1. Drug Description
  • 12.5.2. Regulatory Milestones
  • 12.5.3. Other Developmental Activities
  • 12.5.4. Safety and Efficacy
  • 12.5.5. Product Profile
• 12.6. TIGLUTIK/TEGLUTIK (RILUZOLE): ITF PHARMA (A US SUBSIDIARY OF ITALFARMACO)
  • 12.6.1. Drug Description
  • 12.6.2. Regulatory Milestones
  • 12.6.3. Other Developmental Activities
  • 12.6.4. Safety and Efficacy
  • 12.6.5. Product Profile
• 12.7. RADICAVA/RADICUT (EDARAVONE INJECTION) AND RADICAVA ORS (EDARAVONE ORAL): MITSUBISHI TANABE PHARMA CORPORATION
  • 12.7.1. Drug Description
  • 12.7.2. Regulatory Milestones
  • 12.7.3. Other Developmental Activities
  • 12.7.4. Safety and Efficacy
  • 12.7.5. Product Profile

13. EMERGING DRUGS

• 13.1. KEY COMPETITORS
• 13.2. MASITINIB: AB SCIENCE
  • 13.2.1. Product Description
  • 13.2.2. Other Developmental Activities
  • 13.2.3. Clinical Development
  • 13.2.4. Safety and Efficacy
• 13.3. LATOZINEMAB (AL001/GSK4527223): ALECTOR/GSK
  • 13.3.1. Product Description
  • 13.3.2. Other Developmental Activities
  • 13.3.3. Clinical Development
• 13.4. NUROWN (MSC-NTF CELLS): BRAINSTORM CELL THERAPEUTICS
  • 13.4.1. Product Description
  • 13.4.2. Other Developmental Activities
  • 13.4.3. Clinical Development
  • 13.4.4. Safety and Efficacy
• 13.5. ULEFNERSEN (ION363): IONIS PHARMACEUTICALS
  • 13.5.1. Product Description
  • 13.5.2. Other Developmental Activity
  • 13.5.3. Clinical Development
• 13.6. IBUDILAST: MEDICINOVA
  • 13.6.1. Product Description
  • 13.6.2. Other Developmental Activities
  • 13.6.3. Clinical Development
  • 13.6.4. Safety and Efficacy
• 13.7. DNL343: DENALI THERAPEUTICS
  • 13.7.1. Product Description
  • 13.7.2. Other Developmental Activities
  • 13.7.3. Clinical Development
  • 13.7.4. Safety and Efficacy
• 13.8. ABBV-CLS-7262: ABBVIE/CALICO LIFE SCIENCES
  • 13.8.1. Product Description
  • 13.8.2. Other Developmental Activities
  • 13.8.3. Clinical Development
• 13.9. CNM-AU8: CLENE NANOMEDICINE BIOSCIENCES
  • 13.9.1. Product Description
  • 13.9.2. Other Developmental Activity
  • 13.9.3. Clinical Development
  • 13.9.4. Safety and Efficacy
• 13.1. SLS-005 (TREHALOSE): SEELOS THERAPEUTICS
  • 13.10.1. Product Description
  • 13.10.2. Other Developmental Activities
  • 13.10.3. Clinical Development
  • 13.10.4. Safety and Efficacy
• 13.11. PRIDOPIDINE: PRILENIA THERAPEUTICS
  • 13.11.1. Product Description
  • 13.11.2. Other Developmental Activities
  • 13.11.3. Clinical Development
  • 13.11.4. Safety and Efficacy
• 13.12. RAPA-501: RAPA THERAPEUTICS
  • 13.12.1. Product Description
  • 13.12.2. Other Developmental Activity
  • 13.12.3. Clinical Development
• 13.13. PRIMEC: NEUROSENSE THERAPEUTICS
  • 13.13.1. Product Description
  • 13.13.2. Other Developmental Activities
  • 13.13.3. Clinical Development
  • 13.13.4. Safety and Efficacy
• 13.14. ENGENSIS (VM202): HELIXMITH
  • 13.14.1. Product Description
  • 13.14.2. Other Developmental Activities
  • 13.14.3. Clinical Development
  • 13.14.4. Safety and Efficacy
• 13.15. TPN-101: TRANSPOSON THERAPEUTICS
  • 13.15.1. Product Description
  • 13.15.2. Clinical Development
  • 13.15.3. Safety and Efficacy
• 13.16. RNS60: REVALESIO CORPORATION
  • 13.16.1. Product Description
  • 13.16.2. Other Developmental Activities
  • 13.16.3. Clinical Development
  • 13.16.4. Safety and Efficacy
• 13.17. ANX005: ANNEXON BIOSCIENCES
  • 13.17.1. Product Description
  • 13.17.2. Other Developmental Activity
  • 13.17.3. Clinical Development
  • 13.17.4. Safety and Efficacy
• 13.18. DAZUCORILANT (CORT113176): CORCEPT THERAPEUTICS
  • 13.18.1. Product Description
  • 13.18.2. Other Developmental Activities
  • 13.18.3. Clinical Development
• 13.19. AP-101: AL-S PHARMA
  • 13.19.1. Product Description
  • 13.19.2. Other Developmental Activities
  • 13.19.3. Clinical Development
  • 13.19.4. Safety and Efficacy
• 13.2. SAR443820/DNL788: SANOFI/DENALI THERAPEUTICS
  • 13.20.1. Product Description
  • 13.20.2. Other Developmental Activities
  • 13.20.3. Clinical Development
  • 13.20.4. Safety and Efficacy
• 13.21. AIT-101 (LAM-002A): ORPHAI THERAPEUTICS
  • 13.21.1. Product Description
  • 13.21.2. Other Developmental Activities
  • 13.21.3. Clinical Development
  • 13.21.4. Safety and Efficacy

14. ALS: 7MM ANALYSIS

• 14.1. KEY FINDINGS
• 14.2. MARKET OUTLOOK
• 14.3. CONJOINT ANALYSIS
• 14.4. KEY MARKET FORECAST ASSUMPTIONS
• 14.5. TOTAL MARKET SIZE OF ALS IN THE 7MM
• 14.6. UNITED STATES MARKET SIZE
  • 14.6.1. Total Market Size of ALS in the United States
  • 14.6.2. Market Size of ALS by Therapies in the United States
• 14.7. EU4 AND THE UK MARKET SIZE
  • 14.7.1. Total Market Size of ALS in EU4 and the UK
  • 14.7.2. Market Size of ALS by Therapies in EU4 and the UK
• 14.8. JAPAN MARKET SIZE
  • 14.8.1. Total Market Size of ALS in Japan
  • 14.8.2. Market Size of ALS by Therapies in Japan

15. UNMET NEEDS

16. SWOT ANALYSIS

17. KOL VIEWS

18. MARKET ACCESS AND REIMBURSEMENT

19. APPENDIX

• 19.1. BIBLIOGRAPHY
• 19.2. REPORT METHODOLOGY

20. DELVEINSIGHT CAPABILITIES

21. DISCLAIMER

22. ABOUT DELVEINSIGHT