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.
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 Phase III. 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 CAEL-101, AG10, Amvuttra, and eplontersen, as well as an expected European approval decision for Amvuttra.
- The overall likelihood of approval of a Phase I metabolic-other asset is 8.8%, and the average probability a drug advances from Phase III is 72.2%. Drugs, on average, take 9.0 years from Phase I to approval, compared to 9.7 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 75% of trials in Phase I–II, and 24% 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 Takeda have the highest number of completed clinical trials for amyloidosis, with six trials each.
- GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis, followed by Pfizer.
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
20 PIPELINE DRUGS
25 RECENT EVENTS AND ANALYST OPINION
25 Onpattro for ATTR-CM, Wild Type Or Hereditary (August 3, 2022)
26 Eplontersen for hATTR Amyloidosis With Polyneuropathy (June 21, 2021)
28 AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021)
30 NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
32 Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
35 KEY UPCOMING EVENTS
36 KEY REGULATORY EVENTS
36 CHMP Recommends Approval For Amvuttra
36 Alnylam’s Amvuttra To Take Off From Onpattro’s Runway
36 Alnylam Picks Vutrisiran Extension Over CRL Risk
37 18-Month Data Build Case For Vutrisiran As It Awaits Approval
37 Alnylam’s Vutrisiran Among Myriad New Filings In EU
38 PROBABILITY OF SUCCESS
39 LICENSING AND ASSET ACQUISITION DEALS
39 AstraZeneca Doubles Down On Amyloidosis With Neurimmune Alliance
39 AstraZeneca Goes All In On AL Amyloidosis With Caelum Acquisition
40 REVENUE OPPORTUNITY
42 CLINICAL TRIAL LANDSCAPE
43 Sponsors by status
44 Sponsors by phase
45 Recent events
49 BIBLIOGRAPHY
50 APPENDIX
LIST OF FIGURES
15 Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2017–26
20 Figure 2: Overview of pipeline drugs for amyloidosis in the US
20 Figure 3: Pipeline drugs for amyloidosis, by company
21 Figure 4: Pipeline drugs for amyloidosis, by drug type
21 Figure 5: Pipeline drugs for amyloidosis, by classification
26 Figure 6: Onpattro for ATTR-CM, Wild Type Or Hereditary (August 3, 2022): Phase III – APOLLO-B
30 Figure 7: AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021): Phase III – ATTRibute-CM
32 Figure 8: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021): Phase I – SAD (EU)
34 Figure 9: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021): Phase III – HELIOS A
35 Figure 10: Key upcoming events in amyloidosis
38 Figure 11: Probability of success in the metabolic-other pipeline
42 Figure 12: Clinical trials in amyloidosis
42 Figure 13: Top 10 drugs for clinical trials in amyloidosis
43 Figure 14: Top 10 companies for clinical trials in amyloidosis
43 Figure 15: Trial locations in amyloidosis
44 Figure 16: Amyloidosis trials status
45 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
22 Table 4: Pipeline drugs for amyloidosis in the US
25 Table 5: Onpattro for ATTR-CM, Wild Type Or Hereditary (August 3, 2022)
27 Table 6: Eplontersen for hATTR Amyloidosis With Polyneuropathy (June 21, 2021)
28 Table 7: AG10 for ATTR-CM, Wild Type Or Hereditary (December 27, 2021)
31 Table 8: NTLA-2001 for hATTR Amyloidosis with Polyneuropathy (June 26, 2021)
33 Table 9: Vutrisiran for hATTR Amyloidosis with Polyneuropathy (April 19, 2021)
40 Table 10: Historical global sales, by drug ($m), 2017–21
41 Table 11: Forecasted global sales, by drug ($m), 2022–26
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