$1,318.00
This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key marketed and pipeline drugs, clinical trials, upcoming and regulatory events, recent events and analyst opinion, a 10-year disease incidence forecast, probability of success, and licensing and acquisition deals.
This Market Spotlight report covers the Amyotrophic Lateral Sclerosis (ALS) market, comprising key marketed and pipeline drugs, clinical trials, upcoming and regulatory events, recent events and analyst opinion, a 10-year disease incidence forecast, probability of success, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Key Takeaways
CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
8 Subtypes
9 TREATMENT
9 Symptomatic treatments
9 Ventilatory management
9 Nutritional management
9 Disease-modifying therapies
11 EPIDEMIOLOGY
14 MARKETED DRUGS
16 PIPELINE DRUGS
25 RECENT EVENTS AND ANALYST OPINION
25 CNM-Au8 for ALS (October 3, 2022)
26 Relyvrio for ALS (September 7, 2022)
28 AMX0035 for ALS (March 30, 2022)
31 AMX0035 for ALS (March 28, 2022)
35 CNM-Au8 for ALS (November 2, 2021)
37 IONIS-SOD1Rx for ALS (October 17, 2021)
39 Ultomiris for ALS (August 20, 2021)
40 Miplyffa for ALS (May 7, 2021)
43 KEY UPCOMING EVENTS
44 KEY REGULATORY EVENTS
44 Amylyx’s Relyvrio Could See Fast Sales Growth, But Also Spark Pricing Debate
44 AB Science & Daiichi Try Their Luck Again With EU Filings
45 Second Time’s The Charm: Amylyx’s ALS Drug Wins US FDA Panel Nod
45 Amylyx’s ALS Drug Lacks ‘Confirmatory Evidence’ Needed To Support Approval On Single Study, FDA Says
46 Amylyx ALS Drug: ICER Revised Evidence Report Casts Doubt On Firm’s New Data Analyses
46 Biogen ALS Drug Granted FDA Priority Review, Triggering Aduhelm Déjà Vu
47 Will Amylyx’s ALS Drug Benefit From US FDA’s Aduhelm Experience?
47 US FDA’s ALS Science Strategy Includes Near-Term Rare Neurodegenerative Disease Task Force
48 Mitsubishi’s Oral Radicava Provides Convenience Option For ALS
48 Amylyx Betting On Regulatory Flexibility In ALS
48 Amylyx’s ALS Drug Should Wait For Phase III Results, US FDA Panel Says
49 New EU Filings Include Amylyx’s AMX0035
49 Mitsubishi Tanabe Bids To Take Radicava Oral
50 House ALS Bill Would Require US FDA ‘Action Plan’ – But Not Conditional Approval
50 AMX0035 NDA Rides On CENTAUR, Waits For PEGASUS To Land
51 PROBABILITY OF SUCCESS
52 LICENSING AND ASSET ACQUISITION DEALS
52 NIH Launches Public-Private Partnership For Treating Rare Neurodegenerative Diseases
52 Biogen Returns ALS Drug To Karyopharm
52 Pasithea Therapeutics Acquires Alpha-5 Integrin
53 Pfizer Acquires Biohaven Pharmaceutical For $11.6bn
53 Aquestive Enters Into Licensing Agreement With Haisco
53 Roche Partners With Aquinnah On Oral Therapies For ALS
53 GSK Dives Into Neurology With £30m Oxford University Pact
54 Neumora Launches With $500m To Advance Personalized Neuroscience Drugs
55 REVENUE OPPORTUNITY
56 CLINICAL TRIAL LANDSCAPE
57 Sponsors by status
58 Sponsors by phase
59 Recent events
63 BIBLIOGRAPHY
64 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in incident cases of ALS, 2021–30
16 Figure 2: Overview of pipeline drugs for ALS in the US
16 Figure 3: Pipeline drugs for ALS, by company
17 Figure 4: Pipeline drugs for ALS, by drug type
17 Figure 5: Pipeline drugs for ALS, by classification
26 Figure 6: CNM-Au8 for ALS (October 3, 2022): Phase II/III – HEALEY ALS
37 Figure 7: CNM-Au8 for ALS (November 2, 2021): Phase II – RESCUE-ALS
39 Figure 8: IONIS-SOD1Rx for ALS (October 17, 2021): Phase III – VALOR
42 Figure 9: Miplyffa for ALS (May 7, 2021): Phase III – ORARIALS-01
43 Figure 10: Key upcoming events in ALS
51 Figure 11: Probability of success in the ALS pipeline
56 Figure 12: Clinical trials in ALS
56 Figure 13: Top 10 drugs for clinical trials in ALS
57 Figure 14: Top 10 companies for clinical trials in ALS
57 Figure 15: Trial locations in ALS
58 Figure 16: ALS trials status
59 Figure 17: ALS trials sponsors, by phase
LIST OF TABLES
11 Table 1: Incident cases of ALS, 2021–30
13 Table 2: Incidence rates of ALS, 2021–30
15 Table 3: Marketed drugs for ALS
18 Table 4: Pipeline drugs for ALS in the US
25 Table 5: CNM-Au8 for ALS (October 3, 2022)
26 Table 6: Relyvrio for ALS (September 7, 2022)
29 Table 7: AMX0035 for ALS (March 30, 2022)
32 Table 8: AMX0035 for ALS (March 28, 2022)
36 Table 9: CNM-Au8 for ALS (November 2, 2021)
38 Table 10: IONIS-SOD1Rx for ALS (October 17, 2021)
40 Table 11: Ultomiris for ALS (August 20, 2021)
41 Table 12: Miplyffa for ALS (May 7, 2021)
55 Table 13: Historical global sales, by drug ($m), 2017–21
55 Table 14: Forecasted global sales, by drug ($m), 2022–26
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