表紙
市場調查報告書

到2035年的血友病市場分析和預測

Hemophilia disease forecast and market analysis to 2035

出版商 Datamonitor Healthcare 商品編碼 951553
出版日期 內容資訊 英文 86 Pages
商品交期: 最快1-2個工作天內
價格
到2035年的血友病市場分析和預測 Hemophilia disease forecast and market analysis to 2035
出版日期: 2020年07月14日內容資訊: 英文 86 Pages
簡介

2018年全球診斷出的血友病A人數約為187,750,而血友病B的人數約為35,950。到2027年,預計患病率將分別增加到193,730和38,570。此外,2018年全球範圍內已確診的von Willebrand病(VWD)病例數約為82,550,預計到2027年將增至86,150。迄今為止,全長重組凝血因子VIII產品Advate and Kogenate和凝血因子IX產品BeneFIX佔據了血友病治療領域的主導地位,並佔據了大部分市場份額。但是,這些藥物已被更頻繁地轉換為新一代產品,並且延長的半衰期製劑(EHL)和Hemlibra穩步佔領了市場佔有率。

該報告調查了全球血友病市場,並提供了有關市場趨勢和預測,臨床試驗條件,藥物評估,法規和許可證的信息。

內容

概述

  • 最新要點

疾病背景

  • 定義
  • 患者細分
  • 病因
  • von Willebrand病
  • 血友病症狀
  • VWD症狀
  • 血友病和VWD的併發症
  • 診斷
  • 治療方案

治療

流行病學

  • 患病率調查方法

非處方藥

管道藥物

主要監管事件

  • 基因療法:美國FDA批准用於血友病
  • 基因治療的排他性是兩個主要因素

成功的可能性

許可和資產收購交易

  • uniQure和CSL血友病B治療許可協議
  • 2020年將M&A列為併購目標
  • 羅氏/Spark交易清算FTC,以減輕醫藥交易商的壓力
  • Novo Nordisk和Bluebird基因療法

臨床試驗狀態

  • 按狀態贊助
  • 按階段贊助
  • 最近事件

藥物評估模型

市場動態

趨勢

  • 血友病市場在預測期內不斷擴大
  • 從Hemlibra血友病A領域的替代因素中獲得市場份額
  • 由於管線中的替代凝血促進劑,導致血友病B的市場份額有限
  • 基因治療吸收的限制
  • 血友病A
  • 血友病B

共識預測

最近發生的事件和分析師的看法

  • 針對血友病A的Roctavian(2020年6月17日)
  • Marzeptacog alfa血友病A和B(2019年7月7日)
  • 針對血友病A的Roctavian(2019年5月28日)
  • 針對血友病A的SB-525(2019年4月2日)
  • 針對血友病A的SPK-8011(2019年2月25日)

即將發生的主要事件

主要意見領袖的見識

潛在需求

  • 醫生調查

參考

  • 處方信息

附錄

目錄
Product Code: DMKC0218085

Disease Overview

Hemophilia is a relatively rare hereditary genetic disorder characterized by an inability to produce a clot capable of stopping bleeding. Patients without hemophilia who develop inhibitors against clotting factor VIII or factor IX are diagnosed as having acquired hemophilia A or B, respectively. Another disease associated with blood coagulation dysfunction is von Willebrand disease, which is caused by a deficiency in von Willebrand factor. Although von Willebrand disease is more common than hemophilia, it is relatively less severe.ilia, it is relatively less severe.

Latest Key Takeaways

Datamonitor Healthcare estimates that in 2018, there were approximately 180,750 diagnosed prevalent cases of hemophilia A and 35,950 diagnosed prevalent cases of hemophilia B worldwide. These figures are forecast to increase to 193,730 cases and 38,570 cases, respectively, by 2027. There were approximately 82,550 diagnosed prevalent cases of von Willebrand disease (VWD) worldwide in 2018, which is forecast to increase to 86,150 cases by 2027.

Current treatments are largely focused on replacing factor VIII or IX, the deficiency of which causes hemophilia A or B, respectively. Recombinant factor VIII and IX are at the top of algorithms in the US and EU treatment guidelines. Until now, full-length recombinant factor VIII agents Advate and Kogenate, and the factor IX agent BeneFIX, have dominated the hemophilia space and hold the largest portion of market share. However, these drugs have been steadily losing market share to extended half-life recombinant factor products (EHLs) and Hemlibra, as physicians switch patients to newer-generation products with improved dosing frequencies. In 2019, sales of Advate almost halved due to fierce competition primarily from Roche's Hemlibra, which was awarded a label expansion in the US and EU in the first quarter of 2019.

The therapeutic strategies for hemophilia were revolutionized after the introduction of replacement factors during the late 1990s and early 2000s, which were followed by EHLs over the past decade. Currently, the hemophilia market is undergoing a third revolution with an anticipated shift towards alternative coagulation promoters and gene therapy.

While the 2017 launch of Roche's Hemlibra for the treatment of hemophilia A patients with factor VIII inhibitors did not make a large impact on the hemophilia market, the extension of its US and EU labels in 2018 to include hemophilia A patients without inhibitors triggered a sharp uptake in sales. Hemlibra has captured market share from a host of replacement factors and EHLs, and is set to achieve market-leader status over the forecast period. Hemlibra is clinically more attractive than rival products because of its novel inhibitor-independent mechanism of action, its fortnightly dosing schedule, and its ability to be self-administered subcutaneously in a market dominated by intravenous agents.

There are a number of pipeline candidates in the hemophilia space which may make a significant impact in the market over the forecast period. Based on discussions with key opinion leaders (KOLs), Datamonitor Healthcare expects that Roche's Hemlibra will further extend its lead in the hemophilia space, having captured market-leader status from Takeda's Advate in 2019. Gene therapies are expected to have initially muted uptake because of a high upfront cost for payers ($2m-$3m) and an initial reluctance from prescribers owing to uncertainty over their long-term efficacy and safety.

In the hemophilia B space, pipeline alternative coagulation promoters such as the siRNA agent fitusiran, and the tissue factor pathway inhibitors (TFPIs) concizumab and marstacimab, may struggle to gain uptake due to safety concerns. These drugs meet the need for an effective therapy for hemophilia B patients with inhibitors, provide a more convenient subcutaneous route of administration, and, in the case of fitusiran, provide an impressive once-monthly dosing regimen. However, whether these drugs will come to market is still unclear given substantial safety concerns. While development of fitusiran has continued after a preventable fatality in Phase II trials, continued development of Novo Nordisk's concizumab may be unlikely after three thrombotic adverse events in Phase III trials, representing another setback for the TFPI class. Concerning safety events have not yet been observed in a single Phase I/II study of marstacimab, but the safety of the class as a whole is now in doubt. Given these safety concerns and a lack of physician familiarity, it seems unlikely these drugs will rival recombinant factor IX market leaders BeneFIX and Alprolix for the treatment of hemophilia B patients without inhibitors.

Roctavian (valoctocogene roxaparvovec) is the most advanced gene therapy in the hemophilia A space, and assuming EU and US approvals in Q3 2020, BioMarin is expected to launch the product at a price of $2m-$3m per patient. The extremely high upfront cost of Roctavian will be a significant deterrent to uptake, and we expect its initial use to be limited to severe hemophilia patients on chronic prophylaxis therapy. While gene therapy meets the high unmet need of a long-term therapy, with a projected eight-year interval between doses, Roctavian may face initial payer resistance due to the lack of long-term safety and efficacy data as the follow-up period in current studies has been limited to three years.

TABLE OF CONTENTS

CONTENTS

OVERVIEW

  • Latest key takeaways

DISEASE BACKGROUND

  • Definition
  • Patient segmentation
  • Etiology
  • Von Willebrand disease
  • Hemophilia symptoms
  • VWD symptoms
  • Hemophilia and VWD complications
  • Diagnosis
  • Treatment options

TREATMENT

EPIDEMIOLOGY

  • Prevalence methodology

MARKETED DRUGS

PIPELINE DRUGS

KEY REGULATORY EVENTS

  • Gene Therapies: US FDA Sticks With Bleeding Rate For Hemophilia Approval Endpoint
  • Orphan Exclusivity For Gene Therapies Hinges On Two Big Factors

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

  • uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed
  • Gene Therapy Companies Among Top M&A Targets in 2020
  • Roche/Spark Deal Clears FTC In A Sigh Of Relief For Pharma Dealmakers
  • Novo Nordisk, Bluebird Targeting 'Lifelong' Gene Therapies

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase
  • Recent events

DRUG ASSESSMENT MODEL

MARKET DYNAMICS

FUTURE TRENDS

  • The hemophilia market will continue to expand over the forecast period
  • Hemlibra will continue to capture market share from replacement factors in the hemophilia A space
  • Pipeline alternative coagulation promoters will capture a limited amount of market share in the hemophilia B space
  • Uptake of gene therapy will be limited
  • Hemophilia A
  • Hemophilia B

CONSENSUS FORECASTS

RECENT EVENTS AND ANALYST OPINION

  • Roctavian for Hemophilia A (June 17, 2020)
  • Marzeptacog alfa for Hemophilia A and B (July 7, 2019)
  • Roctavian for Hemophilia A (May 28, 2019)
  • SB-525 for Hemophilia A (April 2, 2019)
  • SPK-8011 for Hemophilia A (February 25, 2019)

KEY UPCOMING EVENTS

KEY OPINION LEADER INSIGHTS

UNMET NEEDS

  • Physician survey

BIBLIOGRAPHY

  • Prescription information

APPENDIX

LIST OF FIGURES

  • Figure 1: Hemophilia etiology
  • Figure 2: Trends in diagnosed prevalent cases of hemophilia A, 2018-27
  • Figure 3: Trends in diagnosed prevalent cases of hemophilia B, 2018-27
  • Figure 4: Trends in diagnosed cases of Von Willebrand disease, 2018-27
  • Figure 5: Overview of pipeline drugs for hemophilia in the US
  • Figure 6: Pipeline drugs for hemophilia, by company
  • Figure 7: Pipeline drugs for hemophilia, by drug type
  • Figure 8: Pipeline drugs for hemophilia, by classification
  • Figure 9: Probability of success in the hemophilia pipeline
  • Figure 10: Clinical trials in hemophilia
  • Figure 11: Top 10 drugs for clinical trials in hemophilia
  • Figure 12: Top 10 companies for clinical trials in hemophilia
  • Figure 13: Trial locations in hemophilia
  • Figure 14: Hemophilia trials status
  • Figure 15: Hemophilia trials sponsors, by phase
  • Figure 16: Datamonitor Healthcare's drug assessment summary for hemophilia
  • Figure 17: Market dynamics in hemophilia (1 of 2)
  • Figure 18: Market dynamics in hemophilia (2 of 2)
  • Figure 19: Future trends in hemophilia
  • Figure 20: Future treatment of hemophilia A - physician survey results
  • Figure 21: Future treatment of hemophilia B - physician survey results
  • Figure 22: Roctavian for Hemophilia A (June 17, 2020) - Phase I/II PoC
  • Figure 23: Marzeptacog alfa for Hemophilia A and B (July 7, 2019) - Phase II/III - MAA-201
  • Figure 24: Key upcoming events in hemophilia
  • Figure 25: Unmet needs in hemophilia, physician survey results

LIST OF TABLES

  • Table 1: Drug classes for hemophilia A, B, and VWD
  • Table 2: MASAC recommendations for hemophilia A, B, and VWD
  • Table 3: Gender distributions of diagnosed prevalent cases of hemophilia A, hemophilia B, and Von Willebrand disease
  • Table 4: Diagnosed prevalent cases of hemophilia A, 2018-27
  • Table 5: Diagnosed prevalent cases of hemophilia B, 2018-27
  • Table 6: Diagnosed prevalent cases of Von Willebrand disease, 2018-27
  • Table 7: Marketed drugs for hemophilia A
  • Table 8: Marketed drugs for hemophilia B
  • Table 9: Marketed drugs for hemophilia A and B
  • Table 10: Marketed drugs for Von Willebrand disease
  • Table 11: Pipeline drugs for hemophilia A
  • Table 12: Pipeline drugs for hemophilia B
  • Table 13: Pipeline drugs for both hemophilia A and B
  • Table 14: Pipeline drugs for Von Willebrand disease
  • Table 15: Historical global sales, by drug ($m), 2015-19
  • Table 16: Forecasted global sales, by drug ($m), 2020-24
  • Table 17: Roctavian for Hemophilia A (June 17, 2020)
  • Table 18: Marzeptacog alfa for Hemophilia A and B (July 7, 2019)
  • Table 19: Roctavian for Hemophilia A (May 28, 2019)
  • Table 20: SB-525 for Hemophilia A (April 2, 2019)
  • Table 21: SPK-8011 for Hemophilia A (February 25, 2019)