DelveInsight’s Achondroplasia pipeline report covers the emerging drugs profiles, including clinical and nonclinical products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type.
Key Highlights from the Achondroplasia Pipeline Report
- There are 5+ drugs in the Achondroplasia pipeline.
- The Achondroplasia pipeline consists of therapies in different clinical phase stages, including Vosoritide, Recifercept, RBM-007, and others.
- Leading players actively working to improve the Achondroplasia treatment landscape are BioMarin, Pfizer, Ribomic Inc., and others.
- Recifercept is a Pfizer asset acquired from Therachon Holdings. In 2019, Pfizer paid USD 360 million for Therachon, a privately-held clinical-stage biotech company, capturing assets such as Recifercept.
- Achondroplasia therapies in the late stage of development include Vosoritide (BioMarin) and others.
- RBM-007 is being developed and invested in the early stage of development by Ribomic Inc.
Get a detailed analysis of the pipeline landscape @ Achondroplasia Pipeline Outlook
Achondroplasia Overview
Achondroplasia is a rare genetic disorder that causes short-limb skeletal dysplasia in about 1 in 20,000-30,000 live births. This genetic disorder is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, which occurs as a result of either a spontaneous genetic mutation (in 80% of patients) or an inherited genetic mutation (in 20% of patients).
Achondroplasia Symptoms
Achondroplasia patients experience the following symptoms:
- Exceptionally large head (macrocephaly)
- Short stature and short upper arms (rhizomelic dwarfism) (adult height of approximately 4 feet)
Achondroplasia Pipeline Analysis: Drug Profiles
Recifercept (TA 46): Pfizer
Product Description
Recifercept (TA 46) is a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy with an MOA are Achondroplasia treatmentsexpected to normalize the overactive FGFR3 signaling pathways that underpin achondroplasia-related bone development abnormalities.
Phase II
NCT04638153: Pfizer launched a Phase II trial titled “A Phase II multiple dose, randomized study to assess the safety, tolerability, pharmacokinetics, and efficacy of Refercept in children with Achondroplasia” in December 2020.
Achondroplasia Pipeline Therapies and Key Companies
- Vosoritide: BioMarin
- Recifercept: Pfizer
- RBM-007: Ribomic Inc.
Find out the emerging therapies and companies @ Achondroplasia Clinical Trials
Achondroplasia Therapeutics Assessment
- By Product Type
- Monotherapy
- Combination Therapy
- By Stage
- Discovery
- Pre-Clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
- By Route of Administration
- Inhalation
- Intravenous
- Oral
- Subcutaneous
- By Molecule Type
- Small Molecule
- Stem Cell Therapy
- Gene Therapy
- Targets
- Immune System
- Multiple Kinase
- By Mechanism of Action
- Protease Inhibitors
- Immunomodulatory
Scope of the Report
Coverage: Global
Achondroplasia Key Players: BioMarin, Pfizer, Ribomic Inc., among others
Achondroplasia Pipeline Therapies: Vosoritide, Recifercept, RBM-007, and others
Table of Contents
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1. |
Introduction |
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2. |
Executive Summary |
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3. |
Achondroplasia: Overview |
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4. |
Achondroplasia – Analytical Perspective In-depth Commercial Assessment |
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5. |
Pipeline Therapeutics |
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6. |
Late Stage Products (Phase III) |
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7. |
Mid Stage Products (Phase II) |
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8. |
Early Stage Products (Phase I) |
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9. |
Pre-clinical and Discovery Stage Products |
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10. |
Therapeutic Assessment |
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11. |
Inactive Products |
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12. |
Company-University Collaborations (Licensing/Partnering) Analysis |
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13. |
Achondroplasia Key Companies |
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14. |
Achondroplasia Key Products |
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15. |
Achondroplasia Unmet Needs |
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16. |
Achondroplasia Market Drivers and Barriers |
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17. |
Achondroplasia Future Perspectives and Conclusion |
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18. |
Achondroplasia Analyst Views |
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19. |
Appendix |
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Key questions answered in the Achondroplasia Pipeline Report
- What Achondroplasia treatments are available?
- How many pharmaceutical companies are working on treatments for Achondroplasia?
- Which of these companies’ pharmaceuticals is the most commonly used?
- How many Achondroplasia medications does each company manufacture?
- How many drugs are in the early, middle, or late stages of development aimed at Achondroplasia?
- How many can current therapies be used alone or in combination with others?
- What are the most significant Achondroplasia collaborations, mergers and acquisitions, and licensing strategies?
Related Reports
DelveInsight’s “Achondroplasia – Market Insights, Epidemiology, and Market Forecast-2030” report delivers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology, and the Achondroplasia market trends in the 7MM.
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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