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Achondroplasia Pipeline Insight, 2019

  • ID: 4835344
  • Drug Pipelines
  • September 2019
  • Region: Global
  • 60 Pages
  • DelveInsight
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FEATURED COMPANIES

  • Ascendis Pharma
  • BioMarin Pharmaceutical
  • BridgeBio Pharma
  • MorphoSys
  • Novartis
  • Novartis
  • MORE
Achondroplasia (Achondroplastic dwarfism)

Overview:

Achondroplasia - Pipeline Insight, 2019 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Achondroplasia pipeline landscape is provided which includes the disease overview and Achondroplasia treatment guidelines. The assessment part of the report embraces, in depth Achondroplasia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Achondroplasia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Achondroplasia Understanding

Achondroplasia also known as Achondroplastic dwarfism is the most common type of short-limbed dwarfism. In achondroplasia the problem is not in forming cartilage but in converting it to bone (a process called ossification), particularly in the long bones of the arms and legs. Achondroplasia is similar to another skeletal disorder called hypochondroplasia, however the features of Achondroplasia tend to be more severe. Achondroplasia is caused by mutations in the FGFR3 gene. People with Achondroplasia generally have normal intelligence levels. Infants born with Achondroplasia typically have a “dome-like” (vaulted) skull, and a very broad forehead. According to National Human Genome Research Institute (NHGRI), in most cases, Achondroplasia isn't inherited, but when it is inherited, it follows an autosomal dominant pattern of inheritance.

Achondroplasia Pipeline Development Activities

The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Achondroplasia targeted therapeutics development with respective active and inactive (dormant or discontinued) projects with the appropriate reasons if available. Achondroplasia pipeline report covers 7+ companies. Some of the key players include BioMarin Pharmaceutical (Vosoritide), Rainier Therapeutics (Vofatamab), Ribomic (RBM 007) etc.

The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.

Achondroplasia Analytical Perspective

In-depth Achondroplasia Commercial Assessment of products

This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition -Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.

Achondroplasia Clinical Assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

Scope of the report
  • The Achondroplasia report provides an overview of therapeutic pipeline activity for Achondroplasia across the complete product development cycle including all clinical and non-clinical stages
  • It comprises of detailed profiles of Achondroplasia therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details
  • Detailed Achondroplasia Research and Development progress and trial details, results wherever available, are also included in the pipeline study
  • Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type
  • Coverage of dormant and discontinued pipeline projects along with the reasons if available across Achondroplasia
Reasons to Buy
  • Establish a comprehensive understanding of the current pipeline scenario across Achondroplasia to formulate effective R&D strategies
  • Assess challenges and opportunities that influence Achondroplasia R&D
  • Develop strategic initiatives by understanding the focus areas of leading companies.
  • Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage
  • Get in detail information of each product with updated information on each project along with key milestones
  • Devise Achondroplasia in licensing and out licensing strategies by identifying prospective partners with progressing projects for Achondroplasia to enhance and expand business potential and scope
  • Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs

Please note: The report will be dispatched within 2-3 days
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Ascendis Pharma
  • BioMarin Pharmaceutical
  • BridgeBio Pharma
  • MorphoSys
  • Novartis
  • Novartis
  • MORE
1. Report Introduction

2. Achondroplasia (Achondroplastic dwarfism)
2.1. Achondroplasia Disease Overview
2.2. Achondroplasia History
2.3. Achondroplasia Symptoms
2.4. Achondroplasia Causes
2.5. Achondroplasia Pathophysiology
2.6. Achondroplasia Diagnosis
2.6.1. Diagnostic Guidelines

3. Achondroplasia Current Treatment Patterns
3.1. Treatment Guidelines

4. Achondroplasia - Analytical Perspective
4.1. In-depth Commercial Assessment
4.1.1. Achondroplasia companies collaborations, Licensing, Acquisition -Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Achondroplasia Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing/Partnering) Analysis
4.1.2.2. Achondroplasia Acquisition Analysis
4.2. Clinical Assessment of Pipeline Drugs
4.2.1. Assessment by Phase of Development
4.2.2. Assessment by Product Type (Mono/Combination)
4.2.2.1. Assessment by Stage and Product Type
4.2.3. Assessment by Route of Administration
4.2.3.1. Assessment by Stage and Route of Administration
4.2.4. Assessment by Molecule Type
4.2.4.1. Assessment by Stage and Molecule Type
4.2.5. Assessment by MOA
4.2.5.1. Assessment by Stage and MOA

5. Achondroplasia Pipeline Therapeutics
5.1. Late Stage Products (Phase-III)
5.1.1. Comparative Analysis
5.2. Mid Stage Products (Phase-II)
5.2.1. Comparative Analysis
5.3. Early Stage Products (Phase-I)
5.3.1. Comparative Analysis
5.4. Pre-clinical and Discovery Stage Products
5.4.1. Comparative Analysis
5.5. Inactive Products

6. Achondroplasia -Products Analysis
6.1. Product Profiles
6.1.1. Vosoritide : BioMarin Pharmaceutical
6.1.1.1. Product Description
6.1.1.1.1. Product Overview
6.1.1.1.2. Mechanism of Action
6.1.1.2. Research and Development
6.1.1.2.1. Clinical Studies
6.1.1.2.1.1. Detailed Study Description
6.1.1.2.1.2. Study Results
6.1.1.2.1.3. Clinical Trials: Tabular View
6.1.1.3. Product Development Activities
6.1.1.3.1. Tabulated Product Summary
6.1.1.3.1.1. General Description Table
6.1.2. Vofatamab: Rainier Therapeutics
6.1.2.1. Product Description
6.1.2.1.1. Product Overview
6.1.2.1.2. Mechanism of Action
6.1.2.2. Research and Development
6.1.2.2.1. Clinical Studies
6.1.2.2.1.1. Detailed Study Description
6.1.2.2.1.2. Study Results
6.1.2.2.1.3. Clinical Trials: Tabular View
6.1.2.3. Product Development Activities
6.1.2.3.1. Tabulated Product Summary
6.1.2.3.1.1. General Description Table
6.1.3. RBM 007: Ribomic
6.1.3.1. Product Description
6.1.3.1.1. Product Overview
6.1.3.1.2. Mechanism of Action
6.1.3.2. Research and Development
6.1.3.2.1. Clinical Studies
6.1.3.2.1.1. Detailed Study Description
6.1.3.2.1.2. Study Results
6.1.3.2.1.3. Clinical Trials: Tabular View
6.1.3.3. Product Development Activities
6.1.3.3.1. Tabulated Product Summary
6.1.3.3.1.1. General Description Table

7. Recent Technologies

8. Achondroplasia Key Companies
8.1. Ascendis Pharma
8.2. BridgeBio Pharma/Novartis
8.3. BioMarin Pharmaceutical
8.4. Pfizer
8.5. Ribomic

9. Achondroplasia Key Products
9.1. TransCon CNP
9.2. Infigratinib
9.3. Vosoritide
9.4. Recifercept
9.5. TA 100
9.6. RBM 007

10. Dormant and Discontinued Products
10.1. Dormant Products
10.1.1. Reasons for being dormant
10.2. Discontinued Products
10.2.1. Reasons for the discontinuation

11. Achondroplasia - Unmet Needs

12. Achondroplasia - Future Perspectives

13. Appendix

14. Report Methodology
14.1. Secondary Research
14.2. Expert Panel Validation
Note: Product cover images may vary from those shown
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  • Ascendis Pharma
  • BridgeBio Pharma
  • Novartis
  • BioMarin Pharmaceutical
  • Pfizer
  • Ribomic
  • Rainier Therapeutics
  • MorphoSys
  • ProChon Biotech
  • Novo Nordisk
  • Novartis
Note: Product cover images may vary from those shown
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