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市場調查報告書

市場焦點:類澱粉蛋白沉積症

Market Spotlight: Amyloidosis

出版商 Datamonitor Healthcare 商品編碼 939984
出版日期 內容資訊 英文 43 Pages
商品交期: 最快1-2個工作天內
價格
市場焦點:類澱粉蛋白沉積症 Market Spotlight: Amyloidosis
出版日期: 2020年04月17日內容資訊: 英文 43 Pages
簡介

本報告提供全球類澱粉蛋白沉積症治療藥市場相關調查分析,提供主要的上市藥·開發平台藥物,臨床試驗,最近的趨勢,未來趨勢,成功概率,專利資訊,10年預測等相關的系統性資訊。

目錄

概要

要點

疾病的背景

  • 亞型

治療

  • 化療
  • 標靶治療
  • 手術
  • 骨髓/幹細胞移植
  • 緩和/支持療法

流行病學

  • 類澱粉蛋白沉積症的發生率
  • 澱粉樣變性在多發性骨髓瘤中的發病率
  • TTR類澱粉蛋白沉積症

上市藥

開發平台藥物

最近的趨勢和分析師的見解

不久的將來趨勢

主要的法規趨勢

成功概率

許可證和資產收購交易

本源專利

臨床試驗形勢

  • 贊助商:各狀態
  • 贊助商:不同階段
  • 最近的趨勢

參考文獻

  • 處方資訊

附錄

目錄
Product Code: DMKC0183635

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 and for transthyretin amyloid cardiomyopathy (ATTR-CM), wild type or hereditary. Additionally, Vyndaqel and Vyndamax are approved by the FDA for the treatment of ATTR-CM, wild type or hereditary. Alnylam's Onpattro and Akcea's Tegsedi are approved in the US for hATTR amyloidosis with polyneuropathy in adults. All of these drugs are designed to block the production of TTR amyloid protein.
  • Industry-sponsored drugs in active clinical development for amyloidosis are spread evenly across Phase I and Phase III, with four drugs also in Phase II.
  • 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, myosin, and catechol-O-methyltransferase. 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 and vutrisiran, and topline Phase II trial results for CK-3773274.
  • The overall likelihood of approval of a Phase I metabolic-other asset is 9.9%, and the average probability a drug advances from Phase III is 70.6%. Drugs, on average, take 8.9 years from Phase I to approval in the metabolic-other space, as well as in the overall metabolic space.
  • There have been 11 licensing and asset acquisition deals involving amyloidosis drugs during 2015-20. 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 71% of trials in Phase I-II, and 29% in Phase III-IV.
  • The US has a substantial lead in the number of amyloidosis clinical trials globally. The UK leads the major European markets, while Japan has the top spot in Asia.
  • GlaxoSmithKline has the highest number of completed clinical trials for amyloidosis, with five trials.
  • GlaxoSmithKline leads industry sponsors with the highest overall number of clinical trials for amyloidosis, followed by Pfizer.

TABLE OF CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

  • Subtypes

TREATMENT

  • Chemotherapy
  • Targeted therapy
  • Surgery
  • Bone marrow/stem cell transplantation
  • Palliative or supportive care

EPIDEMIOLOGY

  • Amyloidosis incidence methodology
  • Amyloidosis in multiple myeloma incidence methodology
  • TTR amyloidosis

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

  • NEOD001 for AL Amyloidosis (April 18, 2019)
  • CAEL-101 for AL Amyloidosis (January 31, 2019)
  • AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

  • A Runaway Hit In US, Pfizer Gains EU Nod for Vyndaqel In Cardiomyopathy
  • A Win For Alnylam In England As NICE Reverses Onpattro Rejection
  • Scotland Greenlights More Rare Disease Treatments
  • FDA Clears Vyndamax On Priority Timetable With Vyndaqel

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

  • Alexion Gets Japanese Rights To Eidos ATTR Candidate

PARENT PATENTS

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase
  • Recent events

BIBLIOGRAPHY

  • Prescription information

APPENDIX

LIST OF FIGURES

  • Figure 1: Trends in incident cases of primary amyloidosis in multiple myeloma, 2017-26
  • Figure 2: Overview of pipeline drugs for amyloidosis in the US
  • Figure 3: Pipeline drugs for amyloidosis, by company
  • Figure 4: Pipeline drugs for amyloidosis, by drug type
  • Figure 5: Pipeline drugs for amyloidosis, by classification
  • Figure 6: NEOD001 for AL Amyloidosis (April 18, 2019): Phase III - VITAL
  • Figure 7: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018): Phase II - Dose-Ranging (201)
  • Figure 8: Key upcoming events in amyloidosis
  • Figure 9: Probability of success in the amyloidosis pipeline
  • Figure 10: Licensing and asset acquisition deals in amyloidosis, 2015-20
  • Figure 11: Parent patents in amyloidosis
  • Figure 12: Clinical trials in amyloidosis
  • Figure 13: Top 10 drugs for clinical trials in amyloidosis
  • Figure 14: Top 10 companies for clinical trials in amyloidosis
  • Figure 15: Trial locations in amyloidosis
  • Figure 16: Amyloidosis trials status
  • Figure 17: Amyloidosis trials sponsors, by phase

LIST OF TABLES

  • Table 1: Global incident cases of amyloidosis in adults, by type, 2017-26
  • Table 2: Global incident cases of primary amyloidosis in multiple myeloma, 2017-26
  • Table 3: Marketed drugs for amyloidosis
  • Table 4: Pipeline drugs for amyloidosis in the US
  • Table 5: NEOD001 for AL Amyloidosis (April 18, 2019)
  • Table 6: CAEL-101 for AL Amyloidosis (January 31, 2019)
  • Table 7: AG10 for ATTR-CM, Wild Type or Hereditary (November 10, 2018)