遺傳性血管水腫 (HAE):焦點市場分析

根據Datamonitor Healthcare的估計，在全球遺傳性血管水腫(HAE)的患病病例約154,300件，預計至2028年增加到約168,100件。

美國在世界最進行許多HAE臨床試驗。德國領導主要歐洲市場，以色列在亞洲佔首位。

Takeda (武田藥品工業) ，HAE的臨床試驗的總數是最多的贊助商，其次是Bio Cryst。

本報告提供遺傳性血管水腫 (HAE)的治療藥市場相關分析，在疾病概要和患者數的變化、預測，目前的主要治療方法，現在開發中臨床實驗的進展與上市計劃，主要的治療藥的市場趨勢預測 (今後10年份)，資本交易的動向等調查。

目錄

內容

概要

重要的要點

疾病的背景情況

• 亞型

治療方法

• C1-INH濃縮物
• 血漿來源C1-INH(pdC1-INH)
• 基因改造型C1-INH (RhC1-INH)
• Kallikrein抑制劑
• Bradykinin受體拮抗劑

流行病學

成藥

開發平台藥物

最近的事件和分析師的見解

• HAE的Garadacimab(2020年6月8日)
• HAE的Orladeyo(2020年1月12日)
• HAE的Orladeyo(2019年11月6日)

今後主要的活動

主要的法規活動

• 英國可提前獲得BioCryst的遺傳性血管水腫藥
• Takeda's 申請Firazyr的非專利版的Adalvo
• BioCryst Pharmaceutical的 berotralstat

成功的概率

授權及資產取得的交易

• Pharming從SOBI再取得EU的Ruconest的權利

商機

臨床試驗情勢

• 贊助商:各狀態
• 贊助商:各階段

參考文件

• 處方藥的資訊

附錄

Overview:

This Market Spotlight report covers the Hereditary Angioedema market, comprising key marketed and pipeline drugs, recent events and analyst opinion, clinical trials, upcoming and regulatory events, probability of success, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Datamonitor Healthcare estimates that in 2019, using the most commonly cited prevalence estimate of 1:50,000, there were approximately 154,300 prevalent cases of hereditary angioedema (HAE) worldwide, and forecasts that number to increase to approximately 168,100 prevalent cases by 2028.

The approved drugs in the HAE space target complement pathway, androgen receptors, follicle-stimulating hormone, gonadotropin-releasing hormone, progesterone receptor, bradykinin B2 receptor, and the kinin-kallikrein system. These are commonly administered via the intravenous or subcutaneous routes, with one product being available in an oral formulation.

The majority of industry-sponsored drugs in active clinical development for HAE are in Phase I, with only one drug in the NDA/BLA stage.

Therapies in development for HAE focus on targets such as the kinin-kallikrein system, coagulation factor XII, and bradykinin B2 receptor. The majority of pipeline drugs are administered via the oral route, with the remainder being tested as subcutaneous formulations.

High-impact upcoming events for drugs in the HAE space comprise topline Phase II trial results for KVD900; topline Phase III trial results, an expected CHMP opinion, and an estimated PDUFA date for Orladeyo; and a data exclusivity expiration for Ruconest.

The overall likelihood of approval of a Phase I autoimmune/immunology-other asset is 19.5%, and the average probability a drug advances from Phase III is 68.6%. Drugs, on average, take 9.9 years from Phase I to approval, compared to 9.2 years in the overall autoimmune/immunology space.

The distribution of clinical trials across Phase I-IV indicates that the majority of trials for HAE have been in the early and midphases of development, with 53% of trials in Phase I-II, and 47% in Phase III-IV.

The US has the highest number of HAE clinical trials globally. Germany leads the major European markets, while Israel has the top spot in Asia.

Clinical trial activity in the HAE space is dominated by completed trials. Takeda has the highest number of completed clinical trials for HAE, with 36 trials.

Takeda leads industry sponsors with the highest overall number of clinical trials for HAE, followed by BioCryst.

TABLE OF CONTENTS

CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

• Subtypes

TREATMENT

• C1-INH concentrates
• Plasma-derived C1-INH (pdC1-INH)
• Recombinant C1-INH (rhC1-INH)
• Kallikrein inhibitor
• Bradykinin receptor antagonist

EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

• Garadacimab for HAE (June 8, 2020)
• Orladeyo for HAE (January 12, 2020)
• Orladeyo for HAE (November 6, 2019)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

• UK Early Access For BioCryst's Hereditary Angioedema Drug
• Adalvo Files For Generic Version Of Takeda's Firazyr
• BioCryst Pharmaceuticals' berotralstat Among 14 New Hopefuls At EMA

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

• Pharming Reacquires EU Ruconest Rights From SOBI

REVENUE OPPORTUNITY

CLINICAL TRIAL LANDSCAPE

• Sponsors by status
• Sponsors by phase

BIBLIOGRAPHY

• Prescription information

APPENDIX

LIST OF FIGURES

• Figure 1: Trends in prevalent cases of HAE, 2019-28
• Figure 2: Overview of pipeline drugs for HAE in the US
• Figure 3: Pipeline drugs for HAE, by company
• Figure 4: Pipeline drugs for HAE, by drug type
• Figure 5: Pipeline drugs for HAE, by classification
• Figure 6: Garadacimab for HAE (June 8, 2020): Phase II - CSL312_2001
• Figure 7: Orladeyo for HAE (January 12, 2020): Phase III - APeX-J (Japan)
• Figure 8: Orladeyo for HAE (November 6, 2019): Phase II/III - APeX-S, Phase III - APeX-2
• Figure 9: Key upcoming events in HAE
• Figure 10: Probability of success in the autoimmune/immunology-other pipeline
• Figure 11: Clinical trials in HAE
• Figure 12: Top 10 drugs for clinical trials in HAE
• Figure 13: Top 10 companies for clinical trials in HAE
• Figure 14: Trial locations in HAE
• Figure 15: HAE trials status
• Figure 16: HAE trials sponsors, by phase

LIST OF TABLES

• Table 1: Prevalent cases of HAE, 2019-28
• Table 2: Marketed drugs for HAE
• Table 3: Pipeline drugs for HAE in the US
• Table 4: Garadacimab for HAE (June 8, 2020)
• Table 5: Orladeyo for HAE (January 12, 2020)
• Table 6: Orladeyo for HAE (November 6, 2019)
• Table 7: Historical global sales, by drug ($m), 2015-19
• Table 8: Forecasted global sales, by drug ($m), 2020-24