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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Neurofibromatoses Type I (Von Recklinghausen's Disease) - Drugs In Development, 2022, provides an overview of the Neurofibromatoses Type I (Von Recklinghausen's Disease) (Genetic Disorders) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher's Pharmaceutical and Healthcare latest pipeline guide Neurofibromatoses Type I (Von Recklinghausen's Disease) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Neurofibromatoses Type I (Von Recklinghausen's Disease) (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 (Von Recklinghausen's Disease) (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 Phase III, Phase II, Phase I and Preclinical stages are 1, 8, 5 and 4 respectively. Similarly, the Universities portfolio in Discovery stages comprises 2 molecules, respectively.
Neurofibromatoses Type I (Von Recklinghausen's Disease) (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 the publisher'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 (Von Recklinghausen's Disease) (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Neurofibromatoses Type I (Von Recklinghausen's Disease) (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 (Von Recklinghausen's Disease) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Neurofibromatoses Type I (Von Recklinghausen's Disease) (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 (Von Recklinghausen's Disease) (Genetic Disorders)
Reasons to Buy
- 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 (Von Recklinghausen's Disease) (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 (Von Recklinghausen's Disease) (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.
Table of Contents
- Introduction
- 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
- Advenchen Laboratories LLC
- Alexion Pharmaceuticals Inc
- AstraZeneca Plc
- Binjiang Pharma, Inc.
- Bristol-Myers Squibb Co
- Eli Lilly and Co
- Fochon Pharmaceutical Ltd
- Healx Ltd
- Mulberry Biotherapeutics Inc
- NFlection Therapeutics Inc
- Novartis AG
- Ono Pharmaceutical Co Ltd
- Pasithea Therapeutics Corp
- Pfizer Inc
- Sino Biopharmaceutical Ltd
- SpringWorks Therapeutics Inc
- Vyriad Inc
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Drug Profiles
- abemaciclib - Drug Profile
- Product Description
- Mechanism Of Action
- AL-2846 - Drug Profile
- Product Description
- Mechanism Of Action
- ALXN-1910 - Drug Profile
- Product Description
- Mechanism Of Action
- binimetinib - Drug Profile
- Product Description
- Mechanism Of Action
- CIP-137401 - Drug Profile
- Product Description
- Mechanism Of Action
- FCN-159 - Drug Profile
- Product Description
- Mechanism Of Action
- HL-085 - Drug Profile
- Product Description
- Mechanism Of Action
- ipilimumab - Drug Profile
- Product Description
- Mechanism Of Action
- mirdametinib - Drug Profile
- Product Description
- Mechanism Of Action
- MUL-001 - Drug Profile
- Product Description
- Mechanism Of Action
- Neurofibromatosis type 1 cutaneous neurofibroma (cNF) - Drug Profile
- Product Description
- Mechanism Of Action
- Neurofibromatosis type 1 plexiform neurofibroma (pNF) - Drug Profile
- Product Description
- Mechanism Of Action
- NFX-179 - Drug Profile
- Product Description
- Mechanism Of Action
- nivolumab - Drug Profile
- Product Description
- Mechanism Of Action
- Oncolytic Virus to Target CD46 and SLC5A5 for Oncology - Drug Profile
- Product Description
- Mechanism Of Action
- selumetinib sulfate - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules for Neurofibromatoses Type I - Drug Profile
- Product Description
- Mechanism Of Action
- TQB-3234 - Drug Profile
- Product Description
- Mechanism Of Action
- trametinib dimethyl sulfoxide - Drug Profile
- Product Description
- Mechanism Of Action
- Treatment for Neurofibromatosis Type 1 - Drug Profile
- Product Description
- Mechanism Of Action
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Product Development Milestones
- Featured News & Press Releases
- Sep 27, 2022: Koselugo approved in Japan for paediatric patients with plexiform neurofibromas in neurofibromatosis type 1
- Nov 29, 2021: SpringWorks Therapeutics announces full enrollment of Phase 2b ReNeu trial evaluating Mirdametinib in adult and pediatric patients with NF1-associated plexiform neurofibromas
- Sep 27, 2021: AstraZeneca Pharma India gets DCGI nod to import, market Selumetinib
- Aug 18, 2021: NFlection Therapeutics announces Orphan Drug Designation of NFX-179 for the treatment of cutaneous neurofibromatosis type 1
- Jul 20, 2021: SpringWorks Therapeutics announces issuance of new U.S composition of matter patent to polymorphic form of mirdametinib, extending patent protection into 2041
- Jun 22, 2021: Koselugo approved in the EU for children with neurofibromatosis type 1 and plexiform neurofibromas
- May 31, 2021: AZ’s Koselugo scores regulatory nod for children with rare neurofibroma
- May 26, 2021: NFlection Therapeutics' NFX-179 gel demonstrates safety and significant reduction in p-ERK, a key biomarker driving neurofibroma tumor growth, in positive data from a 28-day phase 2a clinical trial
- Feb 25, 2021: SpringWorks Therapeutics reports interim data from phase 2b ReNeu trial of Mirdametinib for patients with NF1-PN and provides trial update
- Jun 30, 2020: Selumetinib granted orphan drug designation in Japan for neurofibromatosis type 1
- 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
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact Us
- Disclaimer
- Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), 2022
- Number of Products under Development by Companies, 2022
- Number of Products under Development by Universities/Institutes, 2022
- Products under Development by Companies, 2022
- Products under Development by Universities/Institutes, 2022
- Number of Products by Stage and Target, 2022
- Number of Products by Stage and Mechanism of Action, 2022
- Number of Products by Stage and Route of Administration, 2022
- Number of Products by Stage and Molecule Type, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Advenchen Laboratories LLC, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Alexion Pharmaceuticals Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by AstraZeneca Plc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Binjiang Pharma, Inc., 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Bristol-Myers Squibb Co, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Eli Lilly and Co, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Fochon Pharmaceutical Ltd, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Healx Ltd, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Mulberry Biotherapeutics Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by NFlection Therapeutics Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Novartis AG, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Ono Pharmaceutical Co Ltd, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Pasithea Therapeutics Corp, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Pfizer Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Sino Biopharmaceutical Ltd, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by SpringWorks Therapeutics Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Pipeline by Vyriad Inc, 2022
- Neurofibromatoses Type I (Von Recklinghausen’s Disease) - Dormant Projects, 2022
- Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), 2022
- Number of Products under Development by Companies, 2022
- Number of Products by Top 10 Targets, 2022
- Number of Products by Stage and Top 10 Targets, 2022
- Number of Products by Top 10 Mechanism of Actions, 2022
- Number of Products by Stage and Top 10 Mechanism of Actions, 2022
- Number of Products by Top 10 Routes of Administration, 2022
- Number of Products by Stage and Top 10 Routes of Administration, 2022
- Number of Products by Top 10 Molecule Types, 2022
- Number of Products by Stage and Top 10 Molecule Types, 2022
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Advenchen Laboratories LLC
- Alexion Pharmaceuticals Inc
- AstraZeneca Plc
- Binjiang Pharma, Inc.
- Bristol-Myers Squibb Co
- Eli Lilly and Co
- Fochon Pharmaceutical Ltd
- Healx Ltd
- Mulberry Biotherapeutics Inc
- NFlection Therapeutics Inc
- Novartis AG
- Ono Pharmaceutical Co Ltd
- Pasithea Therapeutics Corp
- Pfizer Inc
- Sino Biopharmaceutical Ltd
- SpringWorks Therapeutics Inc
- Vyriad Inc
