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 2021, there were approximately 95,400 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 20,200 incident cases of AL amyloidosis worldwide in 2021.
- Approved drugs in the amyloidosis space target cluster of differentiation 38, the immune system, 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, alpha-synuclein, amyloid beta/amyloid plaques, tau proteins, catechol-O-methyltransferase, coagulation factor IX, CRISPR/CRISPR-Cas9, and B-cell lymphoma 2 (Bcl-2)/Bcl-2 family. 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 eplontersen, Onpattro, vutrisiran, CAEL-101, and AG10, as well as an expected PDUFA date, an estimated CHMP opinion, and European approval decision for vutrisiran.
- The overall likelihood of approval of a Phase I metabolic-other asset is 7.7%, 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 9.8 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 74% of trials in Phase I–II, and 26% 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 Japan has the top spot in Asia.
- Pfizer and Bristol Myers Squibb have the highest number of completed clinical trials for amyloidosis, with five trials each.
- GlaxoSmithKline and Pfizer lead 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
24 RECENT EVENTS AND ANALYST OPINION
24 AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021)
26 NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
28 Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
30 Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021)
33 KEY UPCOMING EVENTS
34 KEY REGULATORY EVENTS
34 Alnylam Picks Vutrisiran Extension Over CRL Risk
34 18-Month Data Build Case For Vutrisiran As It Awaits Approval
34 Alnylam’s Vutrisiran Among Myriad New Filings In EU
35 No To Five, Yes To Three: NICE Decides On Funding For Drugs In England
35 Alnylam’s Vutrisiran NDA Incorporates Earlier Onpattro Trial In hATTR Amyloidosis Patients
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 AstraZeneca Doubles Down On Amyloidosis With Neurimmune Alliance
37 AstraZeneca Goes All In On AL Amyloidosis With Caelum Acquisition
37 Novo Nordisk Looks To Broaden Therapeutic Reach With Move Into ATTR
38 REVENUE OPPORTUNITY
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
47 BIBLIOGRAPHY
48 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
19 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
26 Figure 6: AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021): Phase III – ATTRibute-CM
28 Figure 7: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021): Phase I – SAD (EU)
30 Figure 8: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021): Phase III – HELIOS A
32 Figure 9: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021): Phase III – HELIOS A
33 Figure 10: Key upcoming events in amyloidosis
36 Figure 11: Probability of success in the metabolic-other pipeline
40 Figure 12: Clinical trials in amyloidosis
40 Figure 13: Top 10 drugs for clinical trials in amyloidosis
41 Figure 14: Top 10 companies for clinical trials in amyloidosis
41 Figure 15: Trial locations in amyloidosis
42 Figure 16: Amyloidosis trials status
43 Figure 17: 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
21 Table 4: Pipeline drugs for amyloidosis in the US
24 Table 5: AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021)
26 Table 6: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
28 Table 7: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
30 Table 8: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (January 7, 2021)
38 Table 9: Historical global sales, by drug ($m), 2017–21
39 Table 10: Forecasted global sales, by drug ($m), 2022–26
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