- The author estimates that in 2016, there were 46,080 incident cases of amyotrophic lateral sclerosis (ALS) in adults aged 40 years and older worldwide, and forecasts that number to increase to 54,750 incident cases by 2025.
- Oceania and Europe are estimated to have the highest disease incidence (2.33 and 2.16 cases per 100,000 people, respectively), while Asia and Africa have the lowest incidence (0.62 and 0.89 cases per 100,000 people, respectively).
- Mitsubishi Tanabe’s Radicava (edaravone), Concordia’s Rilutek (riluzole), and Corestem’s Neuronata-R are the only marketed drugs available for ALS.
- A majority of industry-sponsored drugs in active clinical development for ALS are in Phase II. Therapies in mid- and late-stage development for ALS focus on a wide variety of targets. Approximately one third of the drugs in mid-to-late-stage development are administered via the oral route, with the remainder including intravenous, subcutaneous, intrathecal, and intramuscular formulations.
- High-impact upcoming events for drugs in the ALS space include topline Phase I, Phase IIb, and Phase III trial results for BHV0223, H.P. Acthar Gel, NP001, masitinib, and NurOwn, and an expected CHMP opinion for masitinib’s filing.
- There were 17 licensing and asset acquisition activities involving ALS drugs during 2013-17. The $784m strategic collaboration agreement between Biogen and Ionis to develop drugs for the treatment of neurological disorders was the largest deal during this period.
- OR04’s US product patent and Radicava/Radicut’s method-of-use patent are set to expire in 2020, which will open the door to generic entry
- The clinical trials distribution across Phase I-IV indicates that the majority of trials for ALS have been in early and mid-phases of development, with 82% of trials in Phase I-II, and only 18% in Phase III-IV.
- The US has a substantial lead in the number of ALS clinical trials globally. France and the UK lead the major EU markets, while Japan has the top spot in Asia.
- Clinical trial activity in the ALS space is dominated by completed trials. Biogen and Otsuka have the highest number of completed clinical trials for ALS, with 10 completed trials each. Biogen leads industry sponsors with the highest number of Phase I clinical trials for ALS, followed by Otsuka and Roche.
2 DISEASE BACKGROUND
- Symptomatic treatments
- Ventilatory management
- Nutritional management
- Disease-modifying therapies
5 MARKETED DRUGS
- Approvals by country
7 KEY UPCOMING EVENTS
8 KEY REGULATORY EVENTS
- Radicava US Approval Boost To Mitsubishi As Sales Decline
- Deal Watch: Pfizer Begins 2018 With Two Deals, Including ALS Tie-Up With Sangamo
11 REVENUE OPPORTUNITY
12 CLINICAL TRIAL LANDSCAPE
- Sponsors by status
- Sponsors by phase
- Recent Events
- Prescription information
List of Figures
Figure 1: Trends in incident cases of ALS, 2016-25
Figure 2: Incident cases of ALS, 2017
Figure 3: Pipeline drugs for ALS in the US
Figure 4: Pipeline drugs for ALS, by company
Figure 5: Pipeline drugs for ALS, by drug type
Figure 6: Pipeline drugs for ALS, by classifications
Figure 7: Key upcoming events in ALS
Figure 8: Licensing and asset acquisition deals in ALS, 2013-17
Figure 9: Parent patents in ALS
Figure 10: Clinical trials in ALS
Figure 11: Top 10 drugs for clinical trials in ALS
Figure 12: Top 10 companies for clinical trials in ALS
Figure 13: Trial locations in ALS
Figure 14: ALS trials status
Figure 15: ALS trials sponsors, by phase
List of Tables
Table 1: Incident cases of ALS, 2016-25
Table 2: Incidence rates of ALS, 2016-25
Table 3: Marketed drugs for ALS
Table 4: Approvals by country for ALS
Table 5: Pipeline drugs for ALS in the US
Table 6: Historical global sales, by drug ($m), 2012-16
Table 7: Forecasted global sales, by drug ($m), 2017-22