表紙
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 603908
出版日期 內容資訊 英文 46 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: 2019年12月23日內容資訊: 英文 46 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,提供主要的開發平台·上市藥,法規,專利資訊,10年的盛行率預測,專利·取得交易等系統性資訊。

要點

疾病的背景

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

上市藥

  • 核准:各國

開發平台藥物

今後預測的事件

專利·資產取得交易

本源專利

臨床實驗形勢

  • 贊助商:各狀況
  • 贊助商:不同階段等

參考文獻

附錄

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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. Additionally, Vyndaqel and Vyndamax are approved by the FDA for the treatment of transthyretin amyloid cardiomyopathy (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 two drugs also in Phase II.

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, 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. The overall likelihood of approval of a Phase I metabolic-other asset is 9.1%, and the average probability a drug advances from Phase III is 70.6%. Drugs, on average, take 9.2 years from Phase I to approval, compared to 8.8 years in the overall metabolic space.

There have been 11 licensing and asset acquisition deals involving amyloidosis drugs during 2014-19. 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 EU markets, while Japan has the top spot in Asia.

GlaxoSmithKline and Pfizer have the highest number of completed clinical trials for amyloidosis, with four trials each.

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)
  • Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

  • 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
  • Alexion Gets Option To Acquire Caelum Under Co-development Pact
  • Alnylam Inks Onpattro Commercialization Deal In Israel

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: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018): Phase III - ATTR-ACT
  • Figure 9: Key upcoming events in amyloidosis
  • Figure 10: Probability of success in the amyloidosis pipeline
  • Figure 11: Licensing and asset acquisition deals in amyloidosis, 2014-19
  • Figure 12: Parent patents in amyloidosis
  • Figure 13: Clinical trials in amyloidosis
  • Figure 14: Top 10 drugs for clinical trials in amyloidosis
  • Figure 15: Top 10 companies for clinical trials in amyloidosis
  • Figure 16: Trial locations in amyloidosis
  • Figure 17: Amyloidosis trials status
  • Figure 18: 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)
  • Table 8: Vyndaqel for ATTR-CM, Wild Type or Hereditary (August 27, 2018)