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

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Neurofibromatoses Type I Pipeline Drugs Market Report Overview

Neurofibromatosis type 1 (NF1) is also called von Recklinghausen’s disease. It 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 a mutation of the NF1 gene. Symptoms include liver enlargement, glioma, Lisch nodules, and pheochromocytoma. Treatment includes pain medications, surgery, chemotherapy, and radiation therapy.

The NF1 pipeline market research report provides comprehensive information on the therapeutics under development for NF1, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for NF1 and features dormant and discontinued projects.

NF1 Pipeline Products Market Segmentation by Targets

The key targets in the NF1 pipeline products market are Dual Specificity Mitogen Activated Protein Kinase Kinase 1, Dual Specificity Mitogen Activated Protein Kinase Kinase 2, Alkaline Phosphatase Tissue Nonspecific Isozyme, Cells Expressing Membrane Cofactor Protein, Cyclin-Dependent Kinase 4, Cyclin Dependent Kinase 6, and Cytotoxic T Lymphocyte Protein 4 among others. Dual Specificity Mitogen Activated Protein Kinase Kinase 1 is the leading target in the pipeline.

NF1 Pipeline Products Market Analysis by Targets

NF1 Pipeline Products Market Analysis by Targets

For more target insights into the NF1 pipeline products market, download a free report sample

NF1 Pipeline Products Market Segmentation by Mechanisms of Action

The key mechanisms of action in the NF1 pipeline products market are Dual Specificity Mitogen Activated Protein Kinase Kinase 1 Inhibitor, Dual Specificity Mitogen Activated Protein Kinase Kinase 2 Inhibitor, Alkaline Phosphatase Tissue Nonspecific Isozyme Activator, Cyclin Dependent Kinase 4 Inhibitor, Cyclin Dependent Kinase 6 Inhibitor, Cytotoxic T Lymphocyte Protein 4 Antagonist, and Cytotoxic To Cells Expressing Membrane Cofactor Protein among others. Dual Specificity Mitogen Activated Protein Kinase Kinase 1 Inhibitor is the leading MoA in the pipeline market.

NF1 Pipeline Products Market Analysis by Mechanisms of Action

NF1 Pipeline Products Market Analysis by Mechanisms of ActionFor more mechanisms of action insights into the NF1 pipeline products market, download a free report sample

NF1 Pipeline Products Market Segmentation by Routes of Administration

The key routes of administration in the NF1 pipeline products market are oral, intravenous, intraperitoneal, subcutaneous, intralesional, and intrapleural among others. Oral is the most preferred route for administration.

NF1 Pipeline Products Market Analysis by Routes of Administration

NF1 Pipeline Products Market Analysis by Routes of Administration

For more routes of administration insights into the NF1 pipeline products market, download a free report sample

NF1 Pipeline Products Market Segmentation by Molecule Types

The key molecule types in the NF1 pipeline products market are small molecule, monoclonal antibody, biologic, gene therapy, and oncolytic virus. The leading molecule type is the small molecule.

NF1 Pipeline Products Market Analysis by Molecule Types

NF1 Pipeline Products Market Analysis by Molecule TypesFor more molecule type insights into the NF1 pipeline products market, download a free report sample

NF1 Pipeline Products Market - Competitive Landscape

Some of the leading companies in the NF1 pipeline products market are AstraZeneca Plc, Healx Ltd, Advenchen Laboratories LLC, Alexion Pharmaceuticals Inc, Binjiang Pharma, Inc., Bristol-Myers Squibb Co, Eli Lilly and Co, Fochon Pharmaceutical Ltd, and Mulberry Biotherapeutics Inc among others. AstraZeneca Plc is the leading company in the NF1 pipeline products market.

NF1 Pipeline Products Market Analysis by Companies

NF1 Pipeline Products Market Analysis by CompaniesTo know more about the leading players in the NF1 pipeline products market, download a free report sample

NF1 Pipeline Products Market Report Overview

Key Targets Dual Specificity Mitogen Activated Protein Kinase Kinase 1, Dual Specificity Mitogen Activated Protein Kinase Kinase 2, Alkaline Phosphatase Tissue Nonspecific Isozyme, Cells Expressing Membrane Cofactor Protein, Cyclin Dependent Kinase 4, Cyclin Dependent Kinase 6, and Cytotoxic T Lymphocyte Protein 4
Key Mechanisms of Action Dual Specificity Mitogen Activated Protein Kinase Kinase 1 Inhibitor, Dual Specificity Mitogen Activated Protein Kinase Kinase 2 Inhibitor, Alkaline Phosphatase Tissue Nonspecific Isozyme Activator, Cyclin Dependent Kinase 4 Inhibitor, Cyclin Dependent Kinase 6 Inhibitor, Cytotoxic T Lymphocyte Protein 4 Antagonist, and Cytotoxic To Cells Expressing Membrane Cofactor Protein
Key Routes of Administration Oral, Intravenous, Intraperitoneal, Subcutaneous, Intralesional, and Intrapleural
Key Molecule Type Small Molecule, Monoclonal Antibody, Biologic, Gene Therapy, and Oncolytic Virus
Leading Companies AstraZeneca Plc, Healx Ltd, Advenchen Laboratories LLC, Alexion Pharmaceuticals Inc, Binjiang Pharma, Inc., Bristol-Myers Squibb Co, Eli Lilly and Co, Fochon Pharmaceutical Ltd, and Mulberry Biotherapeutics Inc

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of NF1.
  • The pipeline guide reviews pipeline therapeutics for NF1 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 to 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 NF1 therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates NF1 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 the latest news related to pipeline therapeutics for NF1 (Oncology)

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolios and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for NF1.
  • 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 their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding the NF1 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 the pipeline.

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

Table of Contents

Table of Contents

List of Tables

List of Figures

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

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

Table

List of Tables

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 Companies, 2022 (Contd..1)

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

Figures

List of Figures

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

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