Market Spotlight: Amyloidosis
Market Spotlight: Amyloidosis
$1,318.00
This Market Spotlight report covers the Amyloidosis market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, probability of success, licensing and acquisition deals, and a 10-year disease incidence forecast, as well as presenting drug-specific revenue forecasts.
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, upcoming and regulatory events, probability of success, licensing and acquisition deals, and a 10-year disease incidence forecast, as well as presenting drug-specific revenue forecasts.
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 multiple myeloma cases, Datamonitor Healthcare estimates there were approximately 19,300 incident cases of AL amyloidosis worldwide in 2017.
- Approved drugs in the amyloidosis space target cluster of differentiation 38 and transthyretin. These drugs are administered via the intravenous, subcutaneous, and oral routes.
- The largest proportion of industry-sponsored drugs in active clinical development for amyloidosis are in either Phase I or Phase III, with one drug in the NDA/BLA stage.
- Therapies in development for amyloidosis focus on targets such as transthyretin, amyloid – light chain, cluster of differentiation 38, angiogenesis, DNA, alpha-synuclein, amyloid beta/amyloid plaques, tau proteins, catechol-O-methyltransferase, coagulation factor IX, and CRISPR/CRISPR-Cas9. These therapies are administered via the intravenous, oral, and subcutaneous routes.
- High-impact upcoming events for drugs in the amyloidosis space comprise topline Phase III trial results for AG10, Onpattro, vutrisiran, CAEL-101, and eplontersen, as well as an expected PDUFA date and an estimated CHMP opinion for vutrisiran.
- The overall likelihood of approval of a Phase I metabolic-other asset is 8.3%, and the average probability a drug advances from Phase III is 72.2%. Drugs, on average, take 9.1 years from Phase I to approval, compared to 9.6 years in the overall metabolic space.
- 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 73% of trials in Phase I–II, and 27% in Phase III–IV.
- The US leads in terms of the number of amyloidosis clinical trials globally. The UK leads the major European markets, while Israel and Japan share the top spot in Asia.
- GlaxoSmithKline, Pfizer, and Bristol Myers Squibb have the highest number of completed clinical trials for amyloidosis, with five trials each.
- GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis.
CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
8 Subtypes
9 TREATMENT
9 Chemotherapy
9 Targeted therapy
9 Surgery
9 Bone marrow/stem cell transplantation
10 Palliative or supportive care
11 EPIDEMIOLOGY
11 Amyloidosis incidence methodology
12 Amyloidosis in multiple myeloma incidence methodology
15 TTR amyloidosis
17 MARKETED DRUGS
19 PIPELINE DRUGS
25 RECENT EVENTS AND ANALYST OPINION
25 NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
27 Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
29 Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021)
32 KEY UPCOMING EVENTS
33 KEY REGULATORY EVENTS
33 New EU Filings
33 No To Five, Yes To Three: NICE Decides On Funding For Drugs In England
33 Alnylam’s Vutrisiran NDA Incorporates Earlier Onpattro Trial In hATTR Amyloidosis Patients
35 PROBABILITY OF SUCCESS
36 LICENSING AND ASSET ACQUISITION DEALS
36 AstraZeneca Goes All In On AL Amyloidosis With Caelum Acquisition
36 Novo Nordisk Looks To Broaden Therapeutic Reach With Move Into ATTR
37 REVENUE OPPORTUNITY
38 CLINICAL TRIAL LANDSCAPE
39 Sponsors by status
40 Sponsors by phase
41 Recent events
44 BIBLIOGRAPHY
45 APPENDIX
LIST OF FIGURES
15 Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2017–26
19 Figure 2: Overview of pipeline drugs for amyloidosis in the US
20 Figure 3: Pipeline drugs for amyloidosis, by company
20 Figure 4: Pipeline drugs for amyloidosis, by drug type
20 Figure 5: Pipeline drugs for amyloidosis, by classification
27 Figure 6: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021): Phase I – SAD (EU)
29 Figure 7: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021): Phase III – HELIOS A
31 Figure 8: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021): Phase III – HELIOS A
32 Figure 9: Key upcoming events in amyloidosis
35 Figure 10: Probability of success in the metabolic-other pipeline
38 Figure 11: Clinical trials in amyloidosis
38 Figure 12: Top 10 drugs for clinical trials in amyloidosis
39 Figure 13: Top 10 companies for clinical trials in amyloidosis
39 Figure 14: Trial locations in amyloidosis
40 Figure 15: Amyloidosis trials status
41 Figure 16: Amyloidosis trials sponsors, by phase
LIST OF TABLES
12 Table 1: Global incident cases of amyloidosis in adults, by type, 2017–26
14 Table 2: Global incident cases of primary amyloidosis in multiple myeloma, 2017–26
18 Table 3: Marketed drugs for amyloidosis
22 Table 4: Pipeline drugs for amyloidosis in the US
25 Table 5: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
27 Table 6: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
29 Table 7: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021)
37 Table 8: Historical global sales, by drug ($m), 2016–20
37 Table 9: Forecasted global sales, by drug ($m), 2021–25
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