DelveInsight’s, “Achondroplasia – Pipeline Insights, 2022” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Achondroplasia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Achondroplasia: Overview
chondroplasia (also called: ACH, Achondroplasia Dwarfism) is the most commonly occurring abnormality of bone growth (skeletal dysplasia) and is a form of short-limbed dwarfism. The word for word translation of ‘Achondroplasia’ is “without cartilage formation.” Cartilage is a robust but flexible tissue that makes up much of the skeleton during early development. However, in Achondroplasia the problem is not in forming cartilage but in converting it to bone (a process called ossification), especially in the long bones of the arms and legs. This condition is similar to another skeletal disorder called Hypochondroplasia, but the features of Achondroplasia tend to be more severe.
The characteristic features of Achondroplasia include:-
Achondroplasia is caused by a gene alteration (mutation) in the FGFR3 gene. The FGFR3 gene makes a protein called fibroblast growth factor receptor 3 that is involved in converting cartilage to bone. FGFR3 is the only gene known to be associated with Achondroplasia.
The FGFR3 gene provides instructions for making a protein that is involved in the development and maintenance of bone and brain tissue. Two specific mutations in the FGFR3 gene are responsible for almost all cases of Achondroplasia. Researchers believe that these mutations cause the FGFR3 protein to be overly active, which interferes with skeletal development and leads to the disturbances in bone growth seen with this disorder.
In approximately 80% of patients, Achondroplasia occurs as a result of a spontaneous genetic mutation; and in the remaining 20%, it is inherited from a parent.
Currently, there is no way to prevent or treat Achondroplasia, since the majority of cases result from unexpected new mutations.
Treatment with growth hormone does not substantially affect the height of an individual with Achondroplasia. Leg-lengthening surgeries may be considered in some particular cases. Detection of bone abnormalities, particularly in the back, is important to prevent breathing difficulties and leg pain or loss of function. Kyphosis (or hunch-back) may need to be surgically corrected if it does not disappear when the child begins walking.
Surgery may also help in the treatment of bowing of legs. Ear infections need to be treated immediately to avoid the risk of hearing loss. Dental problems may need to be addressed by an orthodontist (a dentist with specialized training in the alignment of teeth).
Children born with Achondroplasia need to have their height, weight, and head circumference monitored using special growth curves standardized for Achondroplasia.Measures to avoid obesity at an early age are recommended.
This segment of the Achondroplasia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I and preclinical. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
BioMarin has developed BMN-111 (vosoritide), an investigational drug derived from a natural human peptide that is a positive regulator of bone growth. Vosoritide binds to a specific receptor, which initiates intracellular signals that inhibit the overactive FGFR3 pathway. The investigational treatment is being studied in children with Achondroplasia under the age of 18 because their bones are still amenable to growth.
Infigratinib (previously known as BGJ398) is an investigational, orally-administered, ATP-competitive, FGFR1-3 tyrosine kinase inhibitor therapy in development for the treatment of patients with FGFR-driven diseases, including cholangiocarcinoma (bile duct cancer), urothelial carcinoma (bladder cancer), and Achondroplasia, a bone growth disorder in children; Infigratinib is not chemotherapy.
QED Therapeutics holds worldwide rights to evaluate its safety and efficacy for multiple FGFR-driven diseases.
TA-46 (Recifercept) is an investigational, soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy, a mechanism of action that is believed to normalize the overactive FGFR3 signaling pathways that underlie bone development abnormalities associated with Achondroplasia. Therachon was developing TA-46 as a weekly subcutaneous injection for children and adolescents living with the condition. However, In May 2019, Pfizer has entered into a definitive agreement to acquire all the shares of Therachon Holding with assets in development for the treatment of Achondroplasia. TA-46 has completed Phase I and has received Orphan Drug Designation from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA).
Preclinical data in the mouse model show the ability of TA-46 to restore bone growth and metabolism defects that are associated with Achondroplasia. The Phase I clinical trial started in 2018 and the first participant has already been dosed. In Phase I clinical trials healthy participants are dosed with the drug (in this case TA-46), so no therapeutic effect is expected from this trial, where the objective is to assess if the drug is safe to administer in a patient and its pharmacokinetics.
PhaseBio Pharmaceuticals is developing CNP-ELP for the treatment of Achondroplasia. At present, it is in the preclinical stage of development. The C-type natriuretic peptide, or CNP, is a regulator of bone growth and can rescue defects in fibroblast growth factor 3 that cause Achondroplasia resulting in dwarfism. Native CNP has a half-life of less than 3 min, limiting its use as a direct therapeutic. CNP-ELP product candidate is being developed to deliver therapeutic levels of CNP with once-weekly subcutaneous injections. In a mouse model, they observed a demonstrated effect on linear growth when the CNP-ELP product candidate was injected once every 4 days.
Further product details are provided in the report……..
This segment of the report provides insights about the different Achondroplasia drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 6+ key companies which are developing the therapies for Achondroplasia. The companies which have their Achondroplasia drug candidates in the most advanced stage, i.e. Phase III include, BioMarin Pharmaceutical.
DelveInsight’s report covers around 5+ products under different phases of clinical development like
Achondroplasia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Products have been categorized under various Molecule types such as
The report provides insights into different therapeutic candidates in phase III, II, I and preclinical stage. It also analyses Achondroplasia therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Achondroplasia drugs.
Current Treatment Scenario and Emerging Therapies:
Introduction
Executive Summary
Achondroplasia: Overview
• Causes
• Mechanism of Action
• Signs and Symptoms
• Diagnosis
• Disease Management
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Achondroplasia – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
• Achondroplasia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Achondroplasia Collaboration Deals
• Company-Company Collaborations (Licensing / Partnering) Analysis
• Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products
• Comparative Analysis
BMN-111: BioMarin Pharmaceutical
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products
• Comparative Analysis
TransCon CNP: Ascendis Pharma
• Product Description
• Research and Development
• Product Development Activities
Infigratinib: QED Therapeutics (BridgeBio)
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products
• Comparative Analysis
TA-46: Pfizer
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
• Comparative Analysis
CNP-ELP: PhaseBio Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Achondroplasia Key Companies
Achondroplasia Key Products
Achondroplasia- Unmet Needs
Achondroplasia- Market Drivers and Barriers
Achondroplasia- Future Perspectives and Conclusion
Achondroplasia Analyst Views
Achondroplasia Key Companies
Appendix
List of Tables
Table 1 Total Products for Achondroplasia
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
List Of Figures
Figure 1 Total Products for Achondroplasia
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
• BioMarin Pharmaceutical
• Ascendis Pharma
• QED Therapeutics (BridgeBio)
• Pfizer
• PhaseBio Pharmaceuticals
• Ribomic
