Achondroplasia - Pipeline Insight, 2024

This “Achondroplasia - Pipeline Insight, 2024” 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.

Geography Covered

• Global coverage

Achondroplasia: Understanding

Achondroplasia: Overview

Achondroplasia is a rare genetic disorder recognized as the most common primary skeletal dysplasia in humans. This form of dysplasia accounts for greater than 90% of cases of disproportionate short stature, also known as dwarfism. Achondroplasia means “without cartilage formation,” and it is categorized as a physeal (growth plate) dysplasia. Achondroplasia results from a point mutation in the gene coding for the transmembrane portion of fibroblast growth factor receptor 3 (FGFR3), which resides on the short arm of chromosome 4. The resultant abnormal chondroid production affects endochondral ossification, resulting in decreased linear bone growth, among other functions. This pathologic process generally spares intramembranous ossification, which takes place in flat bones such as those in the skull (with the exception of the base of the skull), face, and clavicles.

The point mutation arises from two possible base substitutions: a transition of c.1138G>A (guanine to adenine substitution is identified in approximately 98% of affected individuals) and a transversion of c.1138G>C (guanine to cytosine, seen in about 1% of affected individuals). These base substitutions cause the normal GGG codon to change to AGG or CGG, causing glycine to be replaced with arginine (p.Gly380Arg) in both situations, and subsequently affecting the transmembrane domain of FGFR3. Both substitutions confer a pathogenic variant of FGFR3, which leads to a gain-of-function mechanism of FGFR3 and subsequent quantitative growth plate and cartilage defects seen in achondroplasia.

Management of achondroplasia involves an interprofessional team approach, and anticipatory care is essential. Multiple versions of health supervision and treatment guidelines exist for those with achondroplasia, with most including detailed examinations and specific anticipatory guidance for each separate age group. These guidelines should be followed closely by the pediatrician and/or other health practitioners. Close attention should be paid to the development of obesity in these children and monitored to avoid complications later in life.

"Achondroplasia- Pipeline Insight, 2024" 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.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Achondroplasia R&D. The therapies under development are focused on novel approaches to treat/improve Achondroplasia.

Achondroplasia Emerging Drugs Chapters

This segment of the Achondroplasia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Achondroplasia Emerging Drugs

Infigratinib: QED Therapeutics, Inc.

Genetic variants that cause fibroblast growth factor receptor 3 (FGFR3) overactivity can lead to skeletal conditions such as achondroplasia. It is an oral agent under investigation for the potential to improve the irregular bone growth in achondroplasia by directly impacting FGFR3 overactivity, the underlying cause of the condition. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Achondroplasia.

TYRA-300: Tyra Biosciences, Inc.

TYRA-300 is an FGFR3-selective inhibitor agnostic to the gatekeeper mutation, with less hyperphosphatemia mediated by FGFR1 inhibition than pan-FGFR inhibitors in preclinical models. The company is developing TYRA-300 to address long-term complications and improve quality of life in affected individuals with skeletal dysplasias including achondroplasia. Currently, the drug is in the Phase I stage of development to treat Achondroplasia.

Achondroplasia: Therapeutic Assessment

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:

Major Players in Achondroplasia

• There are approx. 5+ 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, QED Therapeutics, Inc.

Phases

This report covers around 5+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Achondroplasia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Achondroplasia therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Achondroplasia drugs.

Achondroplasia Report Insights

• Achondroplasia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Achondroplasia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Achondroplasia drugs?
• How many Achondroplasia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Achondroplasia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Achondroplasia therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Achondroplasia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• QED Therapeutics, Inc.
• Tyra Biosciences, Inc.
• Sanofi

Key Products

• Infigratinib
• TYRA-300
• SAR442501

Please note: The report will be dispatched within 2-3 days