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市場調查報告書

市場焦點:鐮狀細胞貧血症症

Market Spotlight: Sickle Cell Anemia

出版商 Datamonitor Healthcare 商品編碼 573574
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
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市場焦點:鐮狀細胞貧血症症 Market Spotlight: Sickle Cell Anemia
出版日期: 2019年10月31日內容資訊: 英文 46 Pages
簡介

本報告提供鐮狀細胞貧血症症的治療藥市場相關分析,提供疾病概要和患者數的轉變·預測,目前的主要治療方法及臨床實驗·認證情形,鐮狀細胞貧血症症治療藥市場趨勢預測 (今後10年份),資本交易的動向,目前開發中的臨床實驗進展等調查。

分析的要點

疾病的背景情況

治療方法

  • 骨髓移植
  • hydroxycarbamide
  • 輸血
  • 預防的治療
  • 疼痛管理

流行病學

開發平台上的醫藥品

已上市醫藥品

  • 各國的認證情形

今後主要的動向

法規相關的主要動向

  • FDA (美國食品藥物管理局) ,認證Endari的鐮狀細胞貧血的治療藥

授權合約·資產收購交易 (全2件)

母專利

商機

臨床實驗形勢

  • 贊助商一覽:各狀態
  • 贊助商一覽:各期
  • Mast Therapeutics的產品 (第三階段)的臨床實驗失敗

相關分析

  • 處方藥的資訊

附錄

圖表一覽

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目錄
Product Code: DMKC0180558

This Market Spotlight report covers the sickle cell anemia market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming and regulatory events, patent information, probability of success, a 10-year disease prevalence forecast, and licensing and acquisition deals.

Key Takeaways

Datamonitor Healthcare estimates that in 2017, there were 3.2 million prevalent cases of sickle cell anemia worldwide, and forecasts that number to increase to 3.5 million prevalent cases by

2026. Africa is estimated to have had 2.7 million prevalent cases of sickle cell anemia in 2017, accounting for 85% of worldwide cases. The approved drugs in the sickle cell anemia space predominantly target fetal hemoglobin, with the exception of Endari, which targets nicotinamide adenine dinucleotide. These drugs are administered via the oral route.

The largest proportion of industry-sponsored drugs in active clinical development for sickle cell anemia are in Phase I, with two drugs in the NDA/BLA phase.

Therapies in development for sickle cell anemia focus on a wide variety of targets. All the pipeline drugs are administered via the intravenous or oral routes.

High-impact upcoming events for drugs in the sickle cell anemia space comprise an estimated PDUFA date for voxelotor, an expected PDUFA date as well as a CHMP opinion for crizanlizumab, topline Phase I/II trial results for CTX001, and topline Phase II trial results for olinciguat.

The overall likelihood of approval of a Phase I hematology asset is 25.5%, and the average probability a drug advances from Phase III is 73%. Drugs, on average, take 8.9 years from Phase I to approval in the overall hematology space.

There have been 16 licensing and asset acquisition deals involving sickle cell anemia drugs during 2014-19. The $3,520m coexclusive license agreement made in 2017 between Vertex Pharmaceuticals and CRISPR Therapeutics for the development and commercialization of potential hemoglobinopathy treatments, including treatments for sickle cell disease, was the largest deal.

The distribution of clinical trials across Phase I-IV indicates that the majority of trials for sickle cell anemia have been in the early and mid-phases of development, with 80% of trials in Phase I-II, and only 20% in Phase III-IV.

The US has a substantial lead in the number of sickle cell anemia clinical trials globally. The UK leads the major EU markets, while Lebanon has the top spot in Asia.

Clinical trial activity in the sickle cell anemia space is dominated by completed trials. Global Blood Therapeutics and Pfizer have the highest number of completed clinical trials for sickle cell anemia, with nine trials each.

Global Blood Therapeutics leads industry sponsors with the highest overall number of clinical trials for sickle cell anemia, followed by Novartis.

TABLE OF CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

  • Bone marrow transplantation
  • Hydroxyurea
  • Blood transfusions
  • Prophylactic therapy
  • Pain management

EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

  • Rivipansel for Sickle Cell Anemia (August 2, 2019)
  • Olinciguat for Sickle Cell Anemia (April 1, 2019)
  • RVT-1801 for Sickle Cell Anemia (December 3, 2018)
  • Voxelotor for Sickle Cell Anemia (December 3, 2018)
  • Zynteglo for Sickle Cell Anemia (December 3, 2018)
  • BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

  • Emmaus: 'We Might Stand A Better Chance In Europe Without The EMA'
  • Global Blood Therapeutics' Voxelotor On Track For 2020 Sickle Cell Shakeup
  • Global Blood Therapeutics Leaves ASH With Momentum Behind Voxelotor In Sickle Cell Disease

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

  • Emmaus Grants Taiba Rights To Endari In MENA Nations
  • How Brazilian Patients Might Access Sickle Cell Disease Drug Faster

PARENT PATENTS

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase
  • Recent events

BIBLIOGRAPHY

  • Prescription information

APPENDIX

LIST OF FIGURES

  • Figure 1: Overview of pipeline drugs for sickle cell anemia in the US
  • Figure 2: Pipeline drugs for sickle cell anemia, by company
  • Figure 3: Pipeline drugs for sickle cell anemia, by drug type
  • Figure 4: Pipeline drugs for sickle cell anemia, by classification
  • Figure 5: Rivipansel for Sickle Cell Anemia (August 2, 2019): Phase III - RESET
  • Figure 6: Voxelotor for Sickle Cell Anemia (December 3, 2018): Phase III - HOPE
  • Figure 7: Key upcoming events in sickle cell anemia
  • Figure 8: Probability of success in the sickle cell anemia pipeline
  • Figure 9: Licensing and asset acquisition deals in sickle cell anemia, 2014-19
  • Figure 10: Parent patents in sickle cell anemia
  • Figure 11: Clinical trials in sickle cell anemia
  • Figure 12: Top 10 drugs for clinical trials in sickle cell anemia
  • Figure 13: Top 10 companies for clinical trials in sickle cell anemia
  • Figure 14: Trial locations in sickle cell anemia
  • Figure 15: Sickle cell anemia trials status
  • Figure 16: Sickle cell anemia trial sponsors, by phase

LIST OF TABLES

  • Table 1: Prevalent cases of sickle cell anemia, 2017-26
  • Table 2: Marketed drugs for sickle cell anemia
  • Table 3: Pipeline drugs for sickle cell anemia in the US
  • Table 4: Rivipansel for Sickle Cell Anemia (August 2, 2019)
  • Table 5: Olinciguat for Sickle Cell Anemia (April 1, 2019)
  • Table 6: RVT-1801 for Sickle Cell Anemia (December 3, 2018)
  • Table 7: Voxelotor for Sickle Cell Anemia (December 3, 2018)
  • Table 8: Zynteglo for Sickle Cell Anemia (December 3, 2018)
  • Table 9: BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
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