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血友病の市場分析および2035年までの予測

Hemophilia disease forecast and market analysis to 2035

出版日: | 発行: Datamonitor Healthcare | ページ情報: 英文 86 Pages | 納期: 即日から翌営業日

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血友病の市場分析および2035年までの予測
出版日: 2020年07月14日
発行: Datamonitor Healthcare
ページ情報: 英文 86 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 目次
概要

2018年に診断をされた世界の血友病Aの有病者数は約18万750人で、血友病Bの有病者数は約3万5,950人となっています。有病者数は、2027年までにそれぞれ193,730人、38,570人に増加すると予測されています。また、診断されたフォン・ウィレブランド病(VWD)の世界の有病症例件数(2018年)は約8万2,550件であり、2027年にはは8万6,150件に増加すると予測されています。これまで、完全長の組換え第VIII因子製剤であるAdvateとKogenate、および第IX因子製剤であるBeneFIXが、血友病治療領域を支配し、市場シェアの大部分を占めてきました。しかし、これらの薬剤は、投与頻度を向上させた新世代の製品に切り替えられ、半減期延長型製剤(EHL)やHemlibraに着実にシェアを奪われています。

当レポートは、世界の血友病市場について調査しており、市場動向や予測、臨床試験の情勢、薬剤評価、規制やライセンスなどの情報を提供しています。

目次

概要

  • 最新の要点

病気の背景

  • 定義
  • 患者のセグメンテーション
  • 病因
  • フォンヴィレブランド病
  • 血友病の症状
  • VWDの症状
  • 血友病とVWDの合併症
  • 診断
  • 治療の選択肢

治療

疫学

  • 有病率の調査手法

市販薬

パイプライン薬

主な規制イベント

  • 遺伝子治療:米国FDAの血友病の承認
  • 遺伝子治療の孤立した独占権は2つの大きな要因

成功の確率

ライセンスと資産取得取引

  • uniQureとCSLの血友病B治療ライセンス契約
  • 2020年のM&Aターゲットへの遺伝子治療企業のランクイン
  • Roche/Sparkの取引のFTCクリア
  • Novo NordiskとBluebirdの遺伝子治療

臨床試験の情勢

  • ステータス別スポンサー
  • フェーズ別スポンサー
  • 最近の出来事

薬剤評価モデル

市場力学

動向

  • 血友病市場は予測期間にわたる拡大
  • Hemlibraの血友病A分野の代替因子からの市場シェア獲得
  • パイプラインの代替凝固プロモーターによる血友病Bでの限られた市場シェア
  • 遺伝子治療の取込みの制限
  • 血友病A
  • 血友病B

コンセンサス予測

最近のイベントとアナリストの意見

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

主な今後のイベント

主なオピニオンリーダーの洞察

潜在的なニーズ

  • 医師調査

参考文献

  • 処方せん情報

付録

目次
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)
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