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 2016, there were approximately 59,000 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 14,600 cases of AL amyloidosis worldwide in 2016.
- Vyndaqel was the first therapy to gain approval in the EU for TTR-related hereditary amyloidosis (familial amyloid polyneuropathy). The drug is not approved in the US, where it received a complete response letter from the FDA in 2012. Onpattro is the first and only FDA-approved treatment in the US for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is an intravenously administered RNAi therapeutic that utilizes Tekmira’s second-generation lipid nanoparticle technology to target transthyretin (TTR) for the treatment of hATTR amyloidosis.
- The majority of industry-sponsored drugs in active clinical development for amyloidosis are in Phase I or Phase II, with only one drug in the NDA/BLA phase.
- 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, and an estimated PDUFA date.
- The overall likelihood of approval of a Phase I metabolic-other asset is 7.9%, and the average probability a drug advances from Phase III is 62.5%. Drugs, on average, take 9.1 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 distribution of clinical trials 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 number of overall clinical trials for amyloidosis, followed by Pfizer.
CONTENTS
7 OVERVIEW
8 KEY TAKEAWAYS
9 DISEASE BACKGROUND
9 Amyloidosis subtypes
11 TREATMENT
11 Chemotherapy
11 Targeted therapy
11 Surgery
11 Bone marrow/stem cell transplantation
11 Palliative or supportive care
13 EPIDEMIOLOGY
14 Amyloidosis in multiple myeloma incidence methodology
18 MARKETED DRUGS
20 Approvals by country
22 PIPELINE DRUGS
29 RECENT EVENTS AND ANALYST OPINION
29 Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (August 27, 2018)
31 Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 24, 2018)
33 NEOD001 for Amyloid light-chain (AL) Amyloidosis (April 23, 2018)
35 CAEL-101 for Amyloid light-chain (AL) Amyloidosis (April 23, 2018)
37 Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (March 29, 2018)
39 ALN-TTRsc02 for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (March 29, 2018)
40 Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017)
44 Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017)
48 Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (October 16, 2017)
50 Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (September 20, 2017)
53 Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (May 15, 2017)
55 Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 26, 2017)
58 KEY UPCOMING EVENTS
59 PROBABILITY OF SUCCESS
60 LICENSING AND ASSET ACQUISITION DEALS
60 GSK Passes On Taking Inotersen To FDA With Ionis
60 Fortress Subsidiary Licenses Amyloidosis Candidate From Columbia
62 PARENT PATENTS
63 CLINICAL TRIAL LANDSCAPE
64 Sponsors by status
65 Sponsors by phase
66 Recent events
68 BIBLIOGRAPHY
68 Prescription Information
69 APPENDIX
LIST OF FIGURES
17 Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2016–25
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 classifications
31 Figure 6: Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (August 27, 2018): Phase III -ATTR-ACT
33 Figure 7: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 24, 2018) Phase III – ATTRACT: Phase III – APOLLO
35 Figure 8: NEOD001 for Amyloid light-chain (AL) Amyloidosis (April 23, 2018): Phase IIb – PRONTO , Phase IIb – PRONTO (OLE) , Phase III – VITAL
39 Figure 9: Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (March 29, 2018): Phase III – ATTR-ACT
44 Figure 10: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017): Phase II/III – NEURO-TTR
48 Figure 11: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017): Phase III – APOLLO
50 Figure 12: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (October 16, 2017): Phase II/III – NEURO-TTR
53 Figure 13: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (September 20, 2017): Phase III – APOLLO
55 Figure 14: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (May 15, 2017): Trial Data – Top-Line Results
57 Figure 15: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 26, 2017): Phase II – 002 (Ext.)
58 Figure 16: Key upcoming events in amyloidosis
59 Figure 17: Probability of success in the amyloidosis pipeline
60 Figure 18: Licensing and asset acquisition deals in amyloidosis, 2013–18
62 Figure 19: Parent patents in amyloidosis
63 Figure 20: Clinical trials in amyloidosis
63 Figure 21: Top 10 drugs for clinical trials in amyloidosis
64 Figure 22: Top 10 companies for clinical trials in amyloidosis
64 Figure 23: Trial locations in amyloidosis
65 Figure 24: Amyloidosis trials status
66 Figure 25: Amyloidosis trials sponsors, by phase
LIST OF TABLES
14 Table 1: Global incident cases of amyloidosis, by type, 2016–25
16 Table 2: Global incident cases of primary amyloidosis in multiple myeloma, 2016–25
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: Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (August 27, 2018)
32 Table 7: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 24, 2018)
34 Table 8: NEOD001 for Amyloid light-chain (AL) Amyloidosis (April 23, 2018)
36 Table 9: CAEL-101 for Amyloid light-chain (AL) Amyloidosis (April 23, 2018)
37 Table 10: Vyndaqel for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (March 29, 2018)
39 Table 11: ALN-TTRsc02 for Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) (March 29, 2018)
41 Table 12: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017)
45 Table 13: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (November 2, 2017)
49 Table 14: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (October 16, 2017)
51 Table 15: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (September 20, 2017)
54 Table 16: Tegsedi for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (May 15, 2017)
56 Table 17: Onpattro for Hereditary transthyretin (hATTR) amyloidosis with polyneuropathy (April 26, 2017)
© Informa UK Ltd. This document is a licensed product and is not to be reproduced or redistributed
Do you have a subscription to Datamonitor Healthcare, Biomedtracker or Meddevicetracker? You may already have access to these reports, contact your account manager or email pharma@informabi.com for further help or assistance.
