PUBLISHER: DelveInsight | PRODUCT CODE: 1147334
PUBLISHER: DelveInsight | PRODUCT CODE: 1147334
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.
Study Period: 2019-2032.
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.
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).
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.
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.
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.
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.
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.
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 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 (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 (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 (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
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).
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 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) 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 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.
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.
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).
The total market size of ALS in EU-4 is expected to increase during the study period (2019-2032).
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).
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).
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.
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.
The report covers the detailed information of collaborations, acquisition and merger, licensing, patent details and other information for ALS emerging therapies.
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.
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.
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.