【レポートの概要(一部)】
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Phenylketonuria (PKU) Overview 6
Therapeutics Development 7
Pipeline Products for Phenylketonuria (PKU) – Overview 7
Pipeline Products for Phenylketonuria (PKU) – Comparative Analysis 8
Phenylketonuria (PKU) – Therapeutics under Development by Companies 9
Phenylketonuria (PKU) – Pipeline Products Glance 10
Late Stage Products 10
Early Stage Products 11
Phenylketonuria (PKU) – Products under Development by Companies 12
Phenylketonuria (PKU) – Companies Involved in Therapeutics Development 13
BioMarin Pharmaceutical Inc. 13
Codexis, Inc. 14
Erytech Pharma SA 15
Promethera Biosciences S.A. 16
SOM Innovation Biotech SL 17
Phenylketonuria (PKU) – Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Target 19
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Drug Profiles 24
BMN-165 – Drug Profile 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
Cell therapy for Phenylketonuria – Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
HepaStem – Drug Profile 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
Recombinant Enzyme to Replace Phenylalanine Hydroxylase for Phenyketonuria – Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
sapropterin dihydrochloride – Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
SOM-7400 – Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Phenylketonuria (PKU) – Recent Pipeline Updates 32
Phenylketonuria (PKU) – Dormant Projects 35
Phenylketonuria (PKU) – Product Development Milestones 36
Featured News & Press Releases 36
Sep 24, 2014: FDA Receives Paragraph IV Notice Letter for KUVAN (sapropterin dihydrochloride) Tablets 36
Sep 03, 2014: Merck Announces Detailed 26-Week Results from Phase IIIb Study with Kuvan in Children with PKU Below 4 Years of Age 36
Apr 14, 2014: FDA Extends Market Exclusivity Six Months for BioMarin’s Rare Disease Therapy KUVAN (sapropterin dihydrochloride) Powder for Oral Solution and Tablets 38
Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria 39
Feb 19, 2013: BioMarin Announces Kuvan Significantly Improves Inattentiveness In Phenylketonuria Patients 39
Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013 40
Jun 30, 2011: Merck Serono Initiates Phase IIIb European Study SPARK In Children Younger Than Four Years, Suffering From Phenylketonuria 42
Mar 18, 2011: BioMarin Announces Preliminary Results From ADAPT Study 42
Sep 01, 2010: BioMarin Presents Results From Investigator-Sponsored Trials On Kuvan At SSIEM Annual Conference 44
Aug 17, 2010: BioMarin Initiates Phase IIIb Study To Evaluate Effects Of Kuvan On Neurophychiatric Symptoms In Subjects With Phenylketonuria 45
Appendix 46
Methodology 46
Coverage 46
Secondary Research 46
Primary Research 46
Expert Panel Validation 46
Contact Us 47
Disclaimer 47
【レポート販売概要】
■ タイトル:フェニルケトン尿症(Phenylketonuria (PKU))の治療薬パイプライン動向(2015年上半期版)■ 英文:Phenylketonuria (PKU) - Pipeline Review, H1 2015
■ 発行日:2015年1月22日
■ 調査会社:Global Markets Direct
■ 商品コード:GMDHC6110IDB
■ 調査対象地域:グローバル
- モバイルプロジェクターの世界市場2017-2021About Pico ProjectorsPico projector is used to project images through a handheld device. These projectors are embedded into several devices, such as smartphones and cameras, and is popular as pico projectors. These are miniature image, video, image, or personal digital assistant (PDA) projectors and are used for several applications such as projecting information on relatively large screens. Techn …
- 大腸癌治療薬市場:企業動向PharmaPoint: Colorectal Cancer - Current and Future Players Summary GlobalData has released its pharma report, “PharmaPoint: Colorectal Cancer - Current and Future Players”. The report is a vital source of up-to-date information with in-depth analysis on the companies in the rapidly growing Colorectal Cancer Market. The report identifies and analyses the key companies shaping and driving the globa …
- ヒト微生物叢(ヒトマイクロバイオーム)の世界市場予測(~2027年)“Increased focus on human microbiome therapy development to drive the growth of this market.”The global human microbiome market is projected to reach USD 1,731 million in 2027 from 942 million in 2024, at CAGR of 22.5 % during the forecast period. Market growth is largely driven by the increasing focus on the development of human microbiome therapy. It has also become a validated target for drug d …
- 海洋バイオテクノロジーの世界市場:インサイト、市場機会、市場シェア、市場予測Marine biotechnology is generally referred in use of marine organisms, at the whole cell or molecular level which helps in providing technologies, products and processes that will be beneficial for the society. The major factor which drives the global marine biotechnology market is raising demand of consumers towards natural products. The global Marine Biotechnology Market was anticipated to grow …
- 企業向けMラーニング(モバイルラーニング)の世界市場2016-2020:技術的企業向けMラーニング、非技術的企業向けMラーニングAbout Corporate M-Learning Globalization has intensified competition within and outside organizations. As a result, corporates are faced with the challenge of maintaining market leadership and customer base, as well as recruiting and retaining talent. Evolving job roles are demanding new skills, thereby increasing the reliance on external corporate training providers. Consequently, companies are b …
- 電気絶縁材料の世界市場:熱可塑性物質、エポキシ樹脂、セラミックThe electrical insulation materials market is projected to reach USD 9.58 Billion by 2021 at CAGR of 5.82% from 2016. The rising demand for energy has increased the electrical and power transmission and distribution (T&D) activities which has also led to the increased use of electrical insulation materials. The market based on type is segmented into different types of materials such as thermoplast …
- ミッション管理システムの世界市場予測(~2022年)“Mission Management Systems market projected to grow at a CAGR of 11.95% during the forecast period”The mission management systems market is projected to grow from USD 13.97 billion in 2016 to USD 27.50 billion by 2022, at a CAGR of 11.95% from 2016 to 2022. Increasing need for emergency/search & rescue management, situational awareness is expected to drive the market in the coming years. “Based o …
- 日本の病院用品市場(~2021)Japan Hospital Supplies Market Outlook to 2021 - Disposable Hospital Supplies, Mobility Aids and Transportation Equipment, Operating Room Equipment, Patient Examination Devices, Sterilization Equipment and Syringes and Needles Summary GlobalData’s new report, "Japan Hospital Supplies Market Outlook to 2021 - Disposable Hospital Supplies, Mobility Aids and Transportation Equipment, Operating Room E …
- Greenfields Petroleum Corporation:石油・ガスの開発・生産動向及びコスト分析- Q3, 2013Greenfields Petroleum Corporation Oil & Gas Exploration and Production Operations and Cost Analysis - Q3, 2013 Summary Greenfields Petroleum Corporation Oil & Gas Exploration and Production Operations and Cost Analysis is the latest report from GlobalData, the industry analysis specialists, that offers comprehensive information on the operational and financial performance of the company. The repor …
- 世界のBI(ビジネスインテリジェンス)及び分析プラットフォーム市場動向(2014-2018)TechNavio's analysts forecast the Global BI and Analytics Platform market to grow at a CAGR of 8.93 percent over the period 2013-2018. One of the key factors contributing to this market growth is the increasing volume of information generated from enterprise applications. The Global BI and Analytics Platform market has also been witnessing the increasing adoption of SaaS-based BI and analytics pla …
- オートインジェクターの世界市場予測(~2023年):関節リウマチ、多発性硬化症、アナフィラキシー、心臓血管疾患、乾癬、片頭痛The global autoinjectors market is projected to reach USD 85.31 billion by 2023 from USD 28.91 billion in 2018 at a CAGR of 24.2% during the forecast period. The growth of this market is primarily driven by factors such as the growing prevalence of targeted therapies, rising incidence of anaphylaxis, increasing number of regulatory approvals, and the availability of generic versions of autoinjecto …
- 世界のモバイルサービス市場動向(2012-2016)TechNavio's analysts forecast the Global Mobile Services market to grow at a CAGR of 2.26 percent CAGR percent over the period 2012-2016. One of the key factors contributing to this market growth is the increased deployment of next-generation networks. The Global Mobile Services market has also been witnessing an increased adoption of Voice over LTE (VoLTE). However, the reduced use of voice servi …
- 自動車用途以外のディーゼルエンジンの世界市場2017-2021About Diesel Engines Market for Non-Automotive Applications Diesel engines offer better operational performance, which makes them a preferred choice over petrol engines. The market is predicted to experience high growth due to the rising demand for diesel engines from APAC. China, India, South Korea, Vietnam, Indonesia, and Japan are the key countries contributing to market growth in the region. T …
- 世界の防弾服・個人防護市場動向2013-2023:地域別分析"The Global Body Armor and Personal Protection Market 2013-2023 - Country Analysis: Market Profile" provides details of the key markets in each region, offering an analysis of the top segments of Body Armor and Personal Protection, expected to be in demand. It also investigates the top three expected Body Armor and Personal Protection programs, in terms of demand in the key markets in each region.
- 貿易金融の世界市場:製品種類別(サプライチェーンファイナンス、輸出&機関ファイナンス)、サービスプロバイダー別(銀行、貿易金融機関、その他)、エンドユーザー別(輸出業者、輸入業者、貿易業者)The global trade finance market was valued at $39714.2 million in 2018 and is expected to reach $56,065.6 million by 2026, registering a CAGR of 3.79% from 2019 to 2026. Trade finance is the financing of international trade flows, acting as an intermediary between importers and exporters to mitigate the risks involved in transactions and enhance working capital efficiency in businesses. It deals w …