Market Spotlight: Amyloidosis
Market Spotlight: Amyloidosis
$1,318.00
This Market Spotlight report covers the amyloidosis market, comprising key marketed and pipeline drugs, key upcoming events, patent information, a 10-year disease incidence forecast, and licensing and acquisition deals.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
This Market Spotlight report covers the amyloidosis market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, key upcoming events, probability of success, patent information, a 10-year disease incidence forecast, and licensing and acquisition deals.
Key Takeaways
- Datamonitor Healthcare estimates that in 2017, there were approximately 90,500 incident cases of amyloidosis worldwide, with the highest proportion being the primary (amyloid light-chain; AL) amyloidosis type.
- Among incident multiple myeloma cases, Datamonitor Healthcare estimates there were approximately 19,300 cases of AL amyloidosis worldwide in 2017.
- Pfizer’s Vyndaqel was the first therapy to gain approval in the EU for hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. The drug is not approved in the US, where it received a complete response letter from the US Food and Drug Administration (FDA) in 2012. Alnylam’s Onpattro and Akcea’s Tegsedi are FDA-approved treatments in the US for hATTR amyloidosis with polyneuropathy in adults. All of these drugs are designed to block the production of TTR protein.
- The majority of industry-sponsored drugs in active clinical development for amyloidosis are in Phase I. Therapies in development for amyloidosis focus on targets such as transthyretin, amyloid-light chain, cluster of differentiation 38, proteasome, serum amyloid P component, alpha-synuclein, amyloid beta/amyloid plaques, tau proteins, and catechol-Omethyltransferase. These therapies are administered via the intravenous, oral, and subcutaneous routes.
- High-impact upcoming events for drugs in the amyloidosis space comprise topline Phase II trial results for dezamizumab and GSK2315698A.
- The overall likelihood of approval of a Phase I metabolic-other asset is 7.2%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 9.3 years from Phase I to approval, compared to 8.9 years in the overall metabolic space.
- There have been only seven licensing and asset acquisition deals involving amyloidosis drugs during 2013–18. The largest deal was the $1,740m agreement in 2018 between Ionis Pharmaceuticals and Akcea Therapeutics, pursuant to which Akcea was granted an exclusive worldwide license to Ionis’s inotersen and AKCEA-TTR-LRx (formerly IONIS-TTR-LRx).
- The clinical trials distribution across Phase I–IV indicates that the majority of trials for amyloidosis have been in the early and mid-phases of development, with 81% of trials in Phase I–II, and only 19% in Phase III–IV.
- The US has a substantial lead in the number of amyloidosis clinical trials globally, while the UK leads the major EU markets. • GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with four trials.
- GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis, followed by Pfizer.
CONTENTS
7 OVERVIEW
8 KEY TAKEAWAYS
9 DISEASE BACKGROUND
9 Amyloidosis subtypes
10 TREATMENT
10 Chemotherapy
10 Targeted therapy
10 Surgery
10 Bone marrow/stem cell transplantation
10 Palliative or supportive care
12 EPIDEMIOLOGY
12 Amyloidosis incidence methodology
13 Amyloidosis in multiple myeloma incidence methodology
16 TTR amyloidosis
18 MARKETED DRUGS
20 Approvals by country
22 PIPELINE DRUGS
29 RECENT EVENTS AND ANALYST OPINION
29 AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)
31 Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
34 Onpattro for hATTR Amyloidosis with Polyneuropathy (April 24, 2018)
36 NEOD001 for AL Amyloidosis (April 23, 2018)
38 CAEL-101 for AL Amyloidosis (April 23, 2018)
40 Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
42 ALN-TTRsc02 for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
43 Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
47 Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
51 Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017)
53 Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017)
55 Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017)
57 Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017)
60 KEY UPCOMING EVENTS
61 KEY REGULATORY EVENTS
61 US FDA Marches Toward Record Novel Approvals With Ionis’ Tegsedi, Leadiant’s Revcovi
61 Aimovig And Tegsedi In Raft Of Products To Get EMA Thumbs up
62 PROBABILITY OF SUCCESS
63 LICENSING AND ASSET ACQUISITION DEALS
63 GSK Passes On Taking Inotersen To FDA With Ionis
63 Fortress Subsidiary Licenses Amyloidosis Candidate From Columbia
65 PARENT PATENTS
66 CLINICAL TRIAL LANDSCAPE
67 Sponsors by status
68 Sponsors by phase
69 Recent events
71 BIBLIOGRAPHY
71 Prescription information
72 APPENDIX
LIST OF FIGURES
16 Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2017–26
22 Figure 2: Overview of pipeline drugs for amyloidosis in the US
23 Figure 3: Pipeline drugs for amyloidosis, by company
23 Figure 4: Pipeline drugs for amyloidosis, by drug type
23 Figure 5: Pipeline drugs for amyloidosis, by classification
31 Figure 6: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018): Phase II – Dose-Ranging (201)
34 Figure 7: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
36 Figure 8: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 24, 2018): Phase III – APOLLO
38 Figure 9: NEOD001 for AL Amyloidosis (April 23, 2018): Phase IIb – PRONTO, Phase IIb – PRONTO (OLE), Phase III – VITAL
42 Figure 10: Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018): Phase III – ATTR-ACT
47 Figure 11: Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017): Phase II/III – NEURO-TTR
51 Figure 12: Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017): Phase III – APOLLO
53 Figure 13: Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017): Phase II/III – NEURO-TTR
55 Figure 14: Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017): Phase III – APOLLO
57 Figure 15: Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017): Trial Data – Top-Line Results
59 Figure 16: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017): Phase II – 002 (Ext.)
60 Figure 17: Key upcoming events in amyloidosis
62 Figure 18: Probability of success in the amyloidosis pipeline
63 Figure 19: Licensing and asset acquisition deals in amyloidosis, 2013–18
65 Figure 20: Parent patents in amyloidosis
66 Figure 21: Clinical trials in amyloidosis
66 Figure 22: Top 10 drugs for clinical trials in amyloidosis
67 Figure 23: Top 10 companies for clinical trials in amyloidosis
67 Figure 24: Trial locations in amyloidosis
68 Figure 25: Amyloidosis trials status
69 Figure 26: Amyloidosis trials sponsors, by phase
LIST OF TABLES
13 Table 1: Global incident cases of amyloidosis in adults, by type, 2017–26
15 Table 2: Global incident cases of primary amyloidosis in multiple myeloma, 2017–26
19 Table 3: Marketed drugs for amyloidosis
21 Table 4: Approvals by country for amyloidosis
25 Table 5: Pipeline drugs for amyloidosis in the US
29 Table 6: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)
31 Table 7: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)
35 Table 8: Onpattro for hATTR Amyloidosis With Polyneuropathy (April 24, 2018)
37 Table 9: NEOD001 for AL Amyloidosis (April 23, 2018)
39 Table 10: CAEL-101 for AL Amyloidosis (April 23, 2018)
40 Table 11: Vyndaqel for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
42 Table 12: ALN-TTRsc02 for ATTR-CM, Wild Type or Hereditary (March 29, 2018)
44 Table 13: Tegsedi for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
48 Table 14: Onpattro for hATTR Amyloidosis with Polyneuropathy (November 2, 2017)
52 Table 15: Tegsedi for hATTR Amyloidosis with Polyneuropathy (October 16, 2017)
53 Table 16: Onpattro for hATTR Amyloidosis with Polyneuropathy (September 20, 2017)
56 Table 17: Tegsedi for hATTR Amyloidosis with Polyneuropathy (May 15, 2017)
58 Table 18: Onpattro for hATTR Amyloidosis with Polyneuropathy (April 26, 2017)
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