Achondroplasia – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Achondroplasia Market Outlook

Thelansis’s “Achondroplasia Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Achondroplasia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Achondroplasia Overview

Achondroplasia is an autosomal-dominant disorder caused by mutations in the type 3 receptor for a fibroblast growth factor (FGFR3). A range of distinctive physical features characterizes it. Achondroplasia can be suspected during pregnancy in the third trimester when abnormal ultrasound findings, such as limb foreshortening (in the 95th percentile) and a low nasal bridge, become evident. These findings may prompt further intra-uterine imaging. After birth, individuals with achondroplasia display clinical characteristics, including short limbs, especially in the proximal segment, a long trunk, and a narrow thorax. Generally, their birth height is within the normal range. These individuals typically have a large head with frontal bossing, potentially leading to obstetric complications during childbirth. The midface tends to be hypoplastic, indicating an endochondral growth defect at the skull's base. The hands and fingers of individuals with achondroplasia are notably short. In early life, they exhibit a trident appearance due to an inability to fully oppose the third and fourth digits. This condition also features shortened long bones with wide metaphyses, a slim, radiolucent area in the proximal femur, and a horizontal acetabular roof in antenatal radiographic images.

• Achondroplasia arises in about 1 in every 25,000 to 30,000 individuals. That, in turn, translates into around 250,000 affected persons worldwide.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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