시장보고서
상품코드
1147334

루게릭병 시장 : 시장 인사이트, 역학, 시장 예측(-2032년)

Amyotrophic Lateral Sclerosis - Market Insight, Epidemiology And Market Forecast - 2032

발행일: | 리서치사: DelveInsight Business Research LLP | 페이지 정보: 영문 295 Pages | 배송안내 : 1-2일 (영업일 기준)

※ 본 상품은 영문 자료로 한글과 영문 목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문목차를 참고해주시기 바랍니다.

주요 8개국의 루게릭병(ALS) 시장 규모는 2021년에 4억 9,000만 달러로 평가되었습니다. 이 시장은 앞으로도 한층 더 확대될 것으로 예측됩니다.

주요 8개국의 루게릭병(ALS: Amyotrophic Lateral Sclerosis) 시장에 대해 조사했으며, 시장 개요와 함께 역학, 환자 동향, 새로운 치료법, 2032년까지 시장 규모 예측 및 의료 미충족 요구 등의 정보를 제공합니다.

목차

제1장 주요 인사이트

제2장 보고서 서론

제3장 ALS 시장 개요

제4장 역학과 시장 예측 조사 방법

제5장 개요

제6장 주요 이벤트

제7장 SWOT 분석

제8장 질환의 배경과 개요

  • 서론
  • ALS의 유형
  • 원인
  • 위험 요인
  • 증상
  • 임상적 특징
  • 병인
  • 바이오마커
  • 예후 예측
  • 감별 진단
  • 진단

제9장 ALS 치료 및 관리

  • 다학제 통합진료
  • 신경보호 치료
  • 대증요법
  • 적응 및 보조 장비
  • 영양
  • 호흡기 관리
  • 분비물 관리
  • 완화의료 및 임종 케어

제10장 ALS(MALS) 임상 관리에 관한 유럽 신경학회(EFNS)의 가이드라인(2012년)

제11장 미국 신경 컨퍼런스(AAN)의 ALS 환자 케어 및 관리를 위한 가이드라인(2009년)

제12장 ALS 호흡 기능 장애 관리 알고리즘

제13장 일본, 미국, 유럽 ALS 관리 가이드라인

제14장 주요 7개국 및 중국의 역학과 환자수

  • 주요 조사 결과
  • 가정과 근거
  • 주요 7개국 및 중국의 루게릭병 유병률 합계
  • 주요 7개국 및 중국의 루게릭병 진단 환자수
  • 미국
  • EU 4개국 및 영국
  • 일본
  • 중국

제15장 환자 동향

제16장 ALS의 주요 엔드포인트

제17장 출시 약제

제18장 신약

제19장 ALS : 주요 7개국 시장 및 중국 분석

  • 주요 조사 결과
  • 시장 전망
  • 주요 시장 예측의 전제조건
  • 속성 분석
  • 주요 7개국 및 중국의 루게릭병 전체 시장 규모
  • 주요 7개국 및 중국의 루게릭병 시장 규모, 현재 치료법별
  • 주요 7개국 및 중국의 루게릭병 시장 규모, 신흥 치료법별
  • 미국 시장 규모
  • EU 4개국 및 영국 시장 규모
  • 일본 시장 규모
  • 중국 시장 규모

제20장 미충족 요구

제21장 시장 접근과 상환

제22장 부록

제23장 DelveInsight의 서비스 내용

제24장 면책사항

제25장 DelveInsight에 대해

LSH 22.11.11

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, EU-4 (Germany, France, Italy, and Spain), the United Kingdom, Japan and China

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
  • EU-4 (Germany, France, Italy, and Spain), the United Kingdom
  • Japan
  • China

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's 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 Prevalence of ALS, Diagnosed Prevalence of ALS, Gender-specific Distribution of ALS, Mutation-specific Distribution of ALS, Type-specific Distribution of ALS, Distribution based on Site of onset of ALS, Age-specific Distribution of ALS] in the 7MM and China covering the United States, EU-4 countries (Germany, France, Italy, and Spain), the United Kingdom, Japan, and China 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 EU-4 countries (Germany, France, Italy, and Spain), the United Kingdom and Japan in the year 2021.

  • The total diagnosed prevalent cases of ALS in the 7MM and China were 59,130 and 29,095 in 2021, growing at a CAGR of 1.3% and 0.6% respectively during the study period (2019-2032).
  • Epidemiology assessed for the condition showed that the US, in 2021 accounted for approximately 25,817 diagnosed prevalent cases of ALS.
  • Among the EU-4 and the United Kingdom countries in 2021, Germany had the highest diagnosed prevalent cases of ALS with 5,398 cases, followed by UK (4,561) and Italy (4,478). In contrast, Spain had the lowest cases (3,270) in 2021.
  • Japan accounted for 11,183 diagnosed prevalent ALS cases in 2021.
  • In the United States, in 2021, the proportion of male and female cases were 14,948 and 10,869 respectively.
  • As per the analysis, a higher percentage of sporadic ALS was observed in the 7MM and China 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 2,478, 749, 361, 2,189 and 20,039 respectively, in 2021.
  • Japan accounted for approximately 3,892 cases of 70-79 years of age of ALS in 2021.
  • In China, in 2021 the proportion of male and female cases were 17,948 and 11,146 respectively.
  • In 2021, the patients with the spinal site of onset accounted for 21,035 cases, followed by 5,673 cases with the bulbar site of onset, and 2,386 cases with other uncertain regions in China.

ALS Drug Chapters

Drug chapter segment of the ALS report encloses the detailed analysis of ALS marketed drugs and late stage (Phase-III, Phase-II, and Phase I/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. MT-1186 is an oral suspension formulation that contains the same active ingredient as edaravone for IV infusion (Japanese product name: RADICUT injection 30 mg and RADICUT bag for IV infusion 30 mg) for ALS treatment.

In May 2022, the company announced that FDA has approved RADICAVA ORS (edaravone), the oral form of edaravone, for the treatment of ALS, a neurodegenerative disease that currently has no cure and can progress rapidly. RADICAVA ORS offers the same efficacy as RADICAVA (edaravone), an FDA-approved IV treatment shown in a pivotal trial to help slow the loss of physical function in ALS. In March 2022, the company submitted an application to MHLW (Japan) for marketing and manufacturing approval of oral formulation of MT-1186 for treating ALS.

RELYVRIO (AMX0035) Amylyx Pharmaceuticals

RELYVRIO 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. In September 2022, the FDA approved RELYVRIO (sodium phenylbutyrate and taurursodiol) for treating adults with ALS. AMX0035 is an investigational drug not approved for use by EMA; however, MAA was validated for review by EMA'S CHMP in February 2022, and the potential decision is anticipated by 1H 2023.

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. In 2022, TEGLUTIK (riluzole), the only oral suspension for the treatment of Amyotrophic Lateral Sclerosis (ALS), developed and patented by Italfarmaco, has been approved for the Chinese market by the Drug Administration Law of the People's Republic of China.

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

Tofersen (Biogen/Ionis Pharmaceuticals)

Tofersen 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. In Q2 2022, the company presented 12-month data of tofersen in SOD1-ALS from the Phase III VALOR study and it's OLE at the European Network to Cure ALS. In July 2022, the US FDA accepted Biogen's New Drug Application (NDA), filing of tofersen, and an investigational antisense medicine for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS).

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)

Masitinib is an orally administered tyrosine kinase inhibitor, which has already 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. In 2022, the company announced that Health Canada has granted authorization to file a New Drug Submission for Masitinib in treating ALS under the Notice of Compliance with Conditions (NOC/c) policy. In 2022, the company filed an application for conditional Marketing Authorization to the EMA for Alsitek (Masitinib) to treat ALS.

AIT-101 (LAM-002A) - AI Therapeutics

AIT-101 ((LAM-002A) is a proprietary, oral dissolving formulation of a potent and highly selective PIKfyve kinase inhibitor. The active drug substance in AIT-101 is an experimental therapy that has been evaluated and demonstrated to be safe in almost 1,000 human subjects, including healthy volunteers and patients. In 2022, Company announced that it has launched a Phase II clinical trial into AIT-101 (apilimod dimesylate) as a potential oral treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations.

Pridopidine (Prilenia Therapeutics)

Pridopidine is an investigational sigma-1 receptor agonist for ALS treatment. Pridopidine has an established safety profile and therapeutic potential in several neurodegenerative diseases affecting adults and children. In 2022, the company raised an additional USD 10 million, bolstering the Series B financing round and bringing the total capital raised to USD 144 million, pridopidine for patients with Huntington's disease (HD) and ALS through 2024.The data readouts for Prilenia's clinical trials are expected in the next 12 months. The company anticipates sharing data from the HEALEY ALS Platform Trial by late 2022 and PROOF-HD (Huntington's disease) topline results by early 2023.

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.

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. Recently in June 2022, riluzole oral suspension TIGLUTIK was approved in China. In March 2022, Haisco Pharmaceutical acquired the exclusive rights to develop and commercialize EXSERVAN, for the treatment of ALS in China.

The most recent development in ALS was the addition of RELYVRIO for the treatment of ALS. On September 29, 2022, US FDA approved RELYVRIO (sodium phenylbutyrate and taurursodiol) for the treatment of adults with ALS. In May 2022, an oral suspension formulation of RADICAVA was approved by FDA for the treatment of ALS. The oral medication has the same dosing regimen as RADICAVA.

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 includes Tofersen/BIIB067 (Biogen/Ionis Pharmaceuticals), ION363 (Ionis Pharmaceuticals), Ibudilast (MediciNova), Masitinib (AB Science), NurOwn (Brainstorm-Cell Therapeutics), Reldesemtiv (Cytokinetics), Gold Nanocrystals/CNM-Au8 (Clene Nanomedicine), Verdiperstat (Biohaven Pharmaceuticals), and others investigating their candidates for the treatment of ALS in the 7MM and China.

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

Key Findings

  • The market size of ALS in 7MM and China is expected to rise from USD 490 million in 2021 during the study period (2019-2032).
  • Current market dynamics are dominated by the use of RILUTEK, TIGLUTIK, EXSERVAN, NEUDEXTA, RADICAVA, RADICAVA ORS, RELYVRIO, and others (Anti-epileptic drugs, Opioids, NSAIDs, Diuretics, SSRIs, Antidepressants, etc.)
  • The therapies with the potential to get launched in the forecast period include Tofersen, ION363, NurOwn, Masitinib, 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 EU-4 (Germany, Italy, France, and Spain), the United Kingdom, Japan, and China
  • In the United States, the market size of ALS is anticipated to rise from USD 369 million in 2021 during the forecast period.
  • Among the EU-4 countries, Germany had the largest market size (USD 3 million) in 2021, while Spain had the smallest with USD 2 million.
  • In 2021, Japan accounted for a market size of USD 44 million.
  • In 2021, China accounted for a market size of USD 64 million

The United States Market Outlook

The total market size of ALS in the United States is expected to increase with a CAGR of 21.0% in the study period (2019-2032).

EU-4 (Germany, France, Italy, and Spain), the United Kingdom Countries: Market Outlook

The total market size of ALS in EU-4 is expected to increase during 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 20.4% in the study period (2019-2032).

China Market Outlook

The total market size of ALS in the China is expected to increase with a CAGR of 14.4% 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
  • 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
  • There is an ongoing HEALEY ALS platform trial, accelerating the path to new ALS therapies by testing multiple treatments at once, reducing the cost of research by 30%, decreasing the trial time by 50%, and increasing patient participation by 67%
  • Most of the 7MM and China countries possess their own ALS registries, thereby providing a near precise country-wise number for diagnosed prevalent cases of the disease
  • RADICAVA (edaravone) has been an effective therapy for ALS. Recently, oral RADICAVA got approved in the US for the treatment of patients with ALS to increase the patient compliance

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 US (2023) followed by EU-4 and UK (2024) Japan (2025), and China (2026). As per our analysis, Tofersen's drug uptake in 7MM and China is expected to be fast in 7MM and medium- fast in China. Peak share is expected to be 0.9% US, EU-4 0.7%, UK (0.5%), Whereas in japan the peak share is higher approximately 2.6%, in China peak share is approximately 1.4%.

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 and China
  • 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 and China
  • 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, Phase II, and Phase I/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 and China 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 and China during the study period (2019-2032)?
  • What are the key findings pertaining to the market across 7MM and China and which country will have the largest ALS market size during the study period (2019-2032)?
  • At what CAGR, the ALS market is expected to grow in 7MM and China during the study period (2019-2032)?
  • What would be the ALS market outlook across the 7MM and China during the study 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, EU-4 (Germany, Spain, France, and Italy), UK, Japan and China?
  • What is the historical ALS patient pool in seven major markets covering the United States, EU-4 (Germany, Spain, France, and Italy), UK, Japan, and China?
  • What would be the forecasted patient pool of ALS in seven major markets covering the United States, EU-4 (Germany, Spain, France, and Italy), the UK, Japan, and China?
  • Where will be the growth opportunities in the 7MM and China with respect to the patient population pertaining to ALS?
  • Out of all 7MM and China countries, which country would have the highest prevalent population of ALS during the study period (2019-2032)?
  • At what CAGR the patient population is expected to grow in 7MM and China during the study 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 US, Europe, Japan and China?
  • 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, EU-4 (Germany, Spain, Italy, and France), the United Kingdom, Japan, and China
  • 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 marke

Table of Contents

1. Key Insights

2. Report Introduction

3. ALS Market Overview at a Glance

  • 3.1. Market Share (%) Distribution of ALS in 2019 by Countries
  • 3.2. Market Share (%) Distribution of ALS in 2032 by Countries

4. Epidemiology and Market Forecast Methodology

5. Executive Summary

6. Key Events

7. SWOT Analysis

8. Disease Background and Overview

  • 8.1. Introduction
  • 8.2. Types of ALS
    • 8.2.1. Sporadic ALS
    • 8.2.2. Genetic or Familial ALS
  • 8.3. Causes
    • 8.3.1. Genetics
    • 8.3.2. Environmental factors
  • 8.4. Risk factors
  • 8.5. Symptoms
  • 8.6. Clinical Features
    • 8.6.1. Clinical presentation
    • 8.6.2. Amyotrophic lateral sclerosis phenotypes
    • 8.6.3. Subtypes of ALS based on relative UMN versus LMN involvement
    • 8.6.4. Subtypes of motor neuron disease based on the regional distribution of involvement
    • 8.6.5. Subtypes of ALS based on additional frontotemporal involvement
  • 8.7. Pathogenesis
    • 8.7.1. Failure of proteostasis
    • 8.7.2. Disturbed RNA metabolism
    • 8.7.3. Cytoskeletal disturbances and axonal transport defects
  • 8.8. Biomarker
  • 8.9. Prediction of prognosis
  • 8.10. Differential Diagnosis
  • 8.11. Diagnosis
    • 8.11.1. Criteria and requirements for diagnosis

9. Treatment and Management of ALS

  • 9.1. Multidisciplinary Care
  • 9.2. Neuroprotective Treatments
    • 9.2.1. Riluzole
    • 9.2.2. RADICAVA
  • 9.3. Symptomatic Treatments
  • 9.4. Adaptive and Assistive Equipment
  • 9.5. Nutrition
  • 9.6. Respiratory Management
  • 9.7. Secretion Management
  • 9.8. Palliative and End-of-Life Care

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

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

12. Management Algorithm for Respiratory Dysfunction in ALS

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

14. Epidemiology and Patient Population of the 7MM and China

  • 14.1. Key Findings
  • 14.2. Assumptions and Rationale
  • 14.3. Total Prevalent Population of Amyotrophic Lateral Sclerosis in the 7MM and China
  • 14.4. Total Diagnosed Prevalent Population of Amyotrophic Lateral Sclerosis in the 7MM and China
  • 14.5. United States
    • 14.5.1. Prevalence of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.6. Distribution Based on Site of Onset of Amyotrophic Lateral Sclerosis in the United States
    • 14.5.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in the United States
  • 14.6. EU-4 and UK
    • 14.6.1. Prevalence of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.6. Distribution Based on Site of Onset of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 14.6.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
  • 14.7. Japan
    • 14.7.1. Prevalence of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.6. Distribution Based on Site of Onset of Amyotrophic Lateral Sclerosis in Japan
    • 14.7.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in Japan
  • 14.8. China
    • 14.8.1. Prevalence of Amyotrophic Lateral Sclerosis in China
    • 14.8.2. Diagnosed Prevalence of Amyotrophic Lateral Sclerosis in China
    • 14.8.3. Gender-specific Distribution of Amyotrophic Lateral Sclerosis in China
    • 14.8.4. Mutation-specific Distribution of Amyotrophic Lateral Sclerosis in China
    • 14.8.5. Type-specific Distribution of Amyotrophic Lateral Sclerosis in China
    • 14.8.6. Distribution Based on Site of Onset of Amyotrophic Lateral Sclerosis in China
    • 14.8.7. Age-specific Distribution of Amyotrophic Lateral Sclerosis in China

15. Patient Journey

16. Key Endpoints in ALS

17. Marketed Drugs

  • 17.1. Key Cross
  • 17.2. RADICAVA/RADICUT (edaravone injection) and RADICAVA ORS (edaravone oral): Mitsubishi Tanabe Pharma Corporation
    • 17.2.1. Drug description
    • 17.2.2. Product profile
    • 17.2.3. Regulatory milestones
    • 17.2.4. Other developmental activities
    • 17.2.5. Current pipeline activity
    • 17.2.6. Safety and Efficacy
  • 17.3. RELYVRIO: Amylyx Pharmaceuticals
    • 17.3.1. Drug description
    • 17.3.2. Product profile
    • 17.3.3. Regulatory milestones
    • 17.3.4. Current pipeline activity
    • 17.3.5. Safety and efficacy
  • 17.4. EXSERVAN (riluzole oral film): Aquestive Therapeutics
    • 17.4.1. Drug description
    • 17.4.2. Product profile
    • 17.4.3. Regulatory milestones
    • 17.4.4. Other developmental activities
    • 17.4.5. Safety and efficacy
  • 17.5. RILUTEK (riluzole): Sanofi/Covis
    • 17.5.1. Drug description
    • 17.5.2. Product profile
    • 17.5.3. Regulatory milestones
    • 17.5.4. Other developmental activities
    • 17.5.5. Safety and efficacy
  • 17.6. NUEDEXTA (dextromethorphan hydrobromide/quinidine sulfate): Avanir Pharmaceuticals (a subsidiary of Otsuka America)
    • 17.6.1. Drug description
    • 17.6.2. Product profile
    • 17.6.3. Regulatory milestones
    • 17.6.4. Other developmental activities
    • 17.6.5. Safety and efficacy
  • 17.7. TIGLUTIK/ TEGLUTIK (riluzole): ITF Pharma (a US subsidiary of Italfarmaco)
    • 17.7.1. Drug description
    • 17.7.2. Product profile
    • 17.7.3. Regulatory milestones
    • 17.7.4. Other developmental activities
    • 17.7.5. Safety and efficacy

18. Emerging Drugs

  • 18.1. Key Competitors
  • 18.2. Engensis (VM202): Helixmith
    • 18.2.1. Product description
    • 18.2.2. Other developmental activities
    • 18.2.3. Clinical development
    • 18.2.4. Safety and efficacy
  • 18.3. SAR443820/DNL788: Sanofi/Denali Therapeutics
    • 18.3.1. Product description
    • 18.3.2. Other developmental activities
    • 18.3.3. Clinical development
    • 18.3.4. Safety and efficacy
  • 18.4. TPN-101: Transposon Therapeutics
    • 18.4.1. Product description
    • 18.4.2. Clinical development
  • 18.5. Latozinemab (AL001/GSK4527223): Alector/GSK
    • 18.5.1. Product description
    • 18.5.2. Other developmental activities
    • 18.5.3. Clinical development
  • 18.6. PrimeC: NeuroSense Therapeutics
    • 18.6.1. Product description
    • 18.6.2. Other developmental activities
    • 18.6.3. Clinical development
    • 18.6.4. Safety and efficacy
  • 18.7. Tofersen (BIIB067): Biogen/Ionis
    • 18.7.1. Product description
    • 18.7.2. Other developmental activities
    • 18.7.3. Clinical development
    • 18.7.4. Safety and efficacy
  • 18.8. ION363: Ionis Pharmaceuticals
    • 18.8.1. Product description
    • 18.8.2. Other developmental activity
    • 18.8.3. Clinical development
  • 18.9. NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics
    • 18.9.1. Product description
    • 18.9.2. Other developmental activities
    • 18.9.3. Clinical development
    • 18.9.4. Safety and efficacy
  • 18.10. Reldesemtiv: Cytokinetics/Astellas Pharma
    • 18.10.1. Product description
    • 18.10.2. Other developmental activities
    • 18.10.3. Clinical development
    • 18.10.4. Safety and efficacy
  • 18.11. Pegcetacoplan (APL-2): Apellis Pharmaceuticals
    • 18.11.1. Product description
    • 18.11.2. Other developmental activity
    • 18.11.3. Clinical development
  • 18.12. Gold Nanocrystals/CNM-Au8: Clene Nanomedicine Biosciences
    • 18.12.1. Product description
    • 18.12.2. Other developmental activity
    • 18.12.3. Clinical development
    • 18.12.4. Safety and efficacy
  • 18.13. AP-101: AL-S Pharma
    • 18.13.1. Product description
    • 18.13.2. Other developmental activities
    • 18.13.3. Clinical development
    • 18.13.4. Safety and efficacy
  • 18.14. MN-166 (ibudilast): MediciNova
    • 18.14.1. Product description
    • 18.14.2. Other developmental activities
    • 18.14.3. Clinical development
    • 18.14.4. Safety and efficacy
  • 18.15. SLS-005 (trehalose): Seelos Theraputics
    • 18.15.1. Product description
    • 18.15.3. Clinical development
  • 18.16. Pridopidine: Prilenia Therapeutics
    • 18.16.1. Product description
    • 18.16.2. Other developmental activities
    • 18.16.3. Clinical development
    • 18.16.4. Safety and efficacy
  • 18.17. Masitinib: AB Science
    • 18.17.1. Product description
    • 18.17.2. Other developmental activities
    • 18.17.3. Clinical development
    • 18.17.4. Safety and efficacy
  • 18.18. Tegoprubart (AT-1501): Eledon Pharmaceuticals
    • 18.18.1. Product description
    • 18.18.2. Other developmental activities
    • 18.18.3. Clinical development
    • 18.18.4. Safety and efficacy
  • 18.19. RNS60: Revalesio Corporation
    • 18.19.1. Product description
    • 18.19.2. Other developmental activities
    • 18.19.3. Clinical development
    • 18.19.4. Safety and efficacy
  • 18.20. Verdiperstat: Biohaven Pharmaceuticals
    • 18.20.1. Product description
    • 18.20.2. Other developmental activities
    • 18.20.3. Clinical development
    • 18.20.4. Safety and efficacy
  • 18.21. Zilucoplan: UCB Pharma/Ra Pharmaceuticals
    • 18.21.1. Product description
    • 18.21.2. Other developmental activities
    • 18.22.3. Clinical Development

19. ALS: Seven Major Market and China Analysis

  • 19.1. Key Findings
  • 19.2. Market Outlook
  • 19.3. Key Market Forecast Assumptions
  • 19.4. Attribute Analysis
  • 19.5. Total Market Size of Amyotrophic Lateral Sclerosis in the 7MM and China
  • 19.6. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in the 7MM and China
  • 19.7. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in the 7MM and China
  • 19.8. United States Market Size
    • 19.8.1. Total Market Size of Amyotrophic Lateral Sclerosis in the United States
    • 19.8.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in the United States
    • 19.8.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in the United States
  • 19.9. EU-4 and UK Market Size
    • 19.9.1. Total Market size of Amyotrophic Lateral Sclerosis in the EU-4 and the UK
    • 19.9.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in the EU-4 and the UK
    • 19.9.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in the EU-4 and the UK
  • 19.10. Japan Market Size
    • 19.10.1. Total Market size of Amyotrophic Lateral Sclerosis in Japan
    • 19.10.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in Japan
    • 19.10.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in Japan
  • 19.11. China Market Size
    • 19.11.1. Total Market size of Amyotrophic Lateral Sclerosis in China
    • 19.11.2. Market Size of Amyotrophic Lateral Sclerosis by Current Therapies in China
    • 19.11.3. Market Size of Amyotrophic Lateral Sclerosis by Emerging Therapies in China

20. Unmet Needs

21. Market Access and Reimbursement

22. Appendix

  • 22.1. Bibliography
  • 22.2. Report Methodology

23. DelveInsight Capabilities

24. Disclaimer

25. About DelveInsight

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