Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline Review, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline Review, H1 2020
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Neurofibromatoses Type I - Pipeline Review, H1 2020, provides an overview of the Neurofibromatoses Type I (Genetic Disorders) pipeline landscape.
Neurofibromatosis type 1 (NF1), also called von Recklinghausen's disease, is a rare genetic disorder characterized by the development of multiple noncancerous (benign) tumors of nerves and skin (neurofibromas). This is transmitted on chromosome 17 and is caused by mutation of the NF1 gene. Symptoms include liver enlargement, glioma, Lisch nodules and pheochromocytoma. Treatment includes pain medications, surgery, chemotherapy and radiation therapy.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Neurofibromatoses Type I - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Neurofibromatoses Type I (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Neurofibromatoses Type I (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Neurofibromatoses Type I (Von Recklinghausen’s Disease) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II and Phase I stages are 1, 1, 3 and 1 respectively. Similarly, the Universities portfolio in Discovery stages comprises 1 molecules, respectively.
Neurofibromatoses Type I (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Neurofibromatoses Type I - Pipeline Review, H1 2020, provides an overview of the Neurofibromatoses Type I (Genetic Disorders) pipeline landscape.
Neurofibromatosis type 1 (NF1), also called von Recklinghausen's disease, is a rare genetic disorder characterized by the development of multiple noncancerous (benign) tumors of nerves and skin (neurofibromas). This is transmitted on chromosome 17 and is caused by mutation of the NF1 gene. Symptoms include liver enlargement, glioma, Lisch nodules and pheochromocytoma. Treatment includes pain medications, surgery, chemotherapy and radiation therapy.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Neurofibromatoses Type I - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Neurofibromatoses Type I (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Neurofibromatoses Type I (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Neurofibromatoses Type I (Von Recklinghausen’s Disease) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II and Phase I stages are 1, 1, 3 and 1 respectively. Similarly, the Universities portfolio in Discovery stages comprises 1 molecules, respectively.
Neurofibromatoses Type I (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
- The pipeline guide provides a snapshot of the global therapeutic landscape of Neurofibromatoses Type I (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Neurofibromatoses Type I (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Neurofibromatoses Type I (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Neurofibromatoses Type I (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Neurofibromatoses Type I (Genetic Disorders)
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Neurofibromatoses Type I (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Neurofibromatoses Type I (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Introduction
Global Markets Direct Report Coverage
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Overview
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Companies Involved in Therapeutics Development
AstraZeneca Plc
NFlection Therapeutics Inc
Pfizer Inc
SpringWorks Therapeutics Inc
Vyriad Inc
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Drug Profiles
binimetinib - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
mirdametinib - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NFX-179 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oncolytic Virus to Target CD46 and SLC5A5 for Oncology - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
selumetinib sulfate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sirolimus - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Neurofibromatoses Type I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Product Development Milestones
Featured News & Press Releases
Apr 14, 2020: KOSELUGO (selumetinib) approved for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas in pediatric patients, available from Onco360
Apr 13, 2020: FDA approves AstraZeneca's Koselugo to treat rare genetic disorder
Apr 10, 2020: FDA Approves First Ever Treatment for Neurofibromatosis
Mar 18, 2020: In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
Nov 15, 2019: FDA grants priority review to tumour drug selumetinib
Oct 28, 2019: Selumetinib shows clinical benefit in adult patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
Oct 25, 2019: SpringWorks starts Phase IIb trial of mirdametinib in NF1-PN
Jul 30, 2019: European Commission grants Orphan Drug Designation for SpringWorks Therapeutics’ MEK Inhibitor, Mirdametinib, for the treatment of Neurofibromatosis Type 1
Jun 03, 2019: SpringWorks Therapeutics Announces FDA Fast Track Designation for PD-0325901 for the Treatment of a Severe Form of Neurofibromatosis Type 1
Apr 01, 2019: FDA grants AstraZeneca-Merck’s selumetinib breakthrough therapy designation
Dec 10, 2012: Study Identifies Targeted Molecular Therapy For Untreatable NF1 Tumors
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Overview
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Companies Involved in Therapeutics Development
AstraZeneca Plc
NFlection Therapeutics Inc
Pfizer Inc
SpringWorks Therapeutics Inc
Vyriad Inc
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Drug Profiles
binimetinib - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
mirdametinib - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NFX-179 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oncolytic Virus to Target CD46 and SLC5A5 for Oncology - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
selumetinib sulfate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sirolimus - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Neurofibromatoses Type I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Product Development Milestones
Featured News & Press Releases
Apr 14, 2020: KOSELUGO (selumetinib) approved for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas in pediatric patients, available from Onco360
Apr 13, 2020: FDA approves AstraZeneca's Koselugo to treat rare genetic disorder
Apr 10, 2020: FDA Approves First Ever Treatment for Neurofibromatosis
Mar 18, 2020: In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
Nov 15, 2019: FDA grants priority review to tumour drug selumetinib
Oct 28, 2019: Selumetinib shows clinical benefit in adult patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
Oct 25, 2019: SpringWorks starts Phase IIb trial of mirdametinib in NF1-PN
Jul 30, 2019: European Commission grants Orphan Drug Designation for SpringWorks Therapeutics’ MEK Inhibitor, Mirdametinib, for the treatment of Neurofibromatosis Type 1
Jun 03, 2019: SpringWorks Therapeutics Announces FDA Fast Track Designation for PD-0325901 for the Treatment of a Severe Form of Neurofibromatosis Type 1
Apr 01, 2019: FDA grants AstraZeneca-Merck’s selumetinib breakthrough therapy designation
Dec 10, 2012: Study Identifies Targeted Molecular Therapy For Untreatable NF1 Tumors
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by AstraZeneca Plc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by NFlection Therapeutics Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Pfizer Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by SpringWorks Therapeutics Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Vyriad Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects, H1 2020
Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by AstraZeneca Plc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by NFlection Therapeutics Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Pfizer Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by SpringWorks Therapeutics Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Vyriad Inc, H1 2020
Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects, H1 2020
LIST OF FIGURES
Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020
COMPANIES MENTIONED
AstraZeneca Plc
NFlection Therapeutics Inc
Pfizer Inc
SpringWorks Therapeutics Inc
Vyriad Inc
Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020
COMPANIES MENTIONED
AstraZeneca Plc
NFlection Therapeutics Inc
Pfizer Inc
SpringWorks Therapeutics Inc
Vyriad Inc
