Neurofibromatoses Type I (Von Recklinghausen’s Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Neurofibromatoses Type I (Von Recklinghausen’s Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct'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.

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 (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 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 (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.


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
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
AL-2846 – Drug Profile
ALXN-1910 – Drug Profile
binimetinib – Drug Profile
CIP-137401 – Drug Profile
FCN-159 – Drug Profile
HL-085 – Drug Profile
ipilimumab – Drug Profile
mirdametinib – Drug Profile
MUL-001 – Drug Profile
Neurofibromatosis type 1 cutaneous neurofibroma (cNF) – Drug Profile
Neurofibromatosis type 1 plexiform neurofibroma (pNF) – Drug Profile
NFX-179 – Drug Profile
nivolumab – Drug Profile
Oncolytic Virus to Target CD46 and SLC5A5 for Oncology – Drug Profile
selumetinib sulfate – Drug Profile
Small Molecules for Neurofibromatoses Type I – Drug Profile
TQB-3234 – Drug Profile
trametinib dimethyl sulfoxide – Drug Profile
Treatment for Neurofibromatosis Type 1 – Drug Profile
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
List of Tables
Table 1: Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Universities/Institutes, 2022
Table 4: Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022 (Contd..1)
Table 6: Products under Development by Universities/Institutes, 2022
Table 7: Number of Products by Stage and Target, 2022
Table 8: Number of Products by Stage and Mechanism of Action, 2022
Table 9: Number of Products by Stage and Route of Administration, 2022
Table 10: Number of Products by Stage and Molecule Type, 2022
Table 11: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Advenchen Laboratories LLC, 2022
Table 12: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Alexion Pharmaceuticals Inc, 2022
Table 13: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by AstraZeneca Plc, 2022
Table 14: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Binjiang Pharma, Inc., 2022
Table 15: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Bristol-Myers Squibb Co, 2022
Table 16: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Eli Lilly and Co, 2022
Table 17: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Fochon Pharmaceutical Ltd, 2022
Table 18: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Healx Ltd, 2022
Table 19: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Mulberry Biotherapeutics Inc, 2022
Table 20: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by NFlection Therapeutics Inc, 2022
Table 21: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Novartis AG, 2022
Table 22: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Ono Pharmaceutical Co Ltd, 2022
Table 23: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Pasithea Therapeutics Corp, 2022
Table 24: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Pfizer Inc, 2022
Table 25: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Sino Biopharmaceutical Ltd, 2022
Table 26: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by SpringWorks Therapeutics Inc, 2022
Table 27: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Pipeline by Vyriad Inc, 2022
Table 28: Neurofibromatoses Type I (Von Recklinghausen’s Disease) – Dormant Projects, 2022
List of Figures
Figure 1: Number of Products under Development for Neurofibromatoses Type I (Von Recklinghausen’s Disease), 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Top 10 Targets, 2022
Figure 4: Number of Products by Stage and Top 10 Targets, 2022
Figure 5: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Mechanism of Actions, 2022
Figure 7: Number of Products by Top 10 Routes of Administration, 2022
Figure 8: Number of Products by Stage and Top 10 Routes of Administration, 2022
Figure 9: Number of Products by Molecule Types, 2022
Figure 10: Number of Products by Stage and Molecule Types, 2022

Download our eBook: How to Succeed Using Market Research

Learn how to effectively navigate the market research process to help guide your organization on the journey to success.

Download eBook
Cookie Settings