Press release from ABNewswire
The Key Achondroplasia Companies in the market include – BridgeBio/ QED Therapeutics , Ribomic Inc, Sanofi, Changchun GeneScience Pharma, Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others.
DelveInsight’s “Achondroplasia Market Insights, Epidemiology, and Market Forecast-2034? report offers an in-depth understanding of the Achondroplasia, historical and forecasted epidemiology as well as the Achondroplasia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
To Know in detail about the Achondroplasia market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Achondroplasia Market Forecast
Some of the key facts of the Achondroplasia Market Report:
- The Achondroplasia market size is anticipated to grow with a significant CAGR during the study period (2020-2034)
- In November 2024, RIBOMIC, Inc. (TOKYO:4591), a clinical-stage pharmaceutical company focused on aptamer therapeutics, has been conducting a Phase IIa clinical trial of umedaptanib pegol (anti-FGF2 aptamer) in pediatric patients (ages 5-14) with achondroplasia (ACH). The company announced today that the low-dose (0.3 mg/kg) subcutaneous injection group (cohort 1), administered once a week, has completed treatment, showing a positive effect on the growth rate of the patients.
- In September 2024, BridgeBio Pharma, Inc. (Nasdaq: BBIO), a biopharmaceutical company specializing in genetic diseases, today announced that the FDA has granted Breakthrough Therapy Designation to oral infigratinib, which is being developed for the treatment of children with achondroplasia.
- In Febraury 2024, Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company dedicated to developing next-generation precision medicines targeting Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to TYRA-300, an oral FGFR3 selective inhibitor, for the treatment of achondroplasia.
- In the United States, achondroplasia impacts a slightly higher percentage of females (51.2%) compared to males (48.8%). This slight difference may be attributed to genetic or biological factors that contribute to its somewhat greater prevalence in females.
- In 2023, the total number of diagnosed prevalent cases of achondroplasia in Japan and the US was approximately 18,000, with expectations for this number to increase in the coming years.
- In 2023, Japan had around 3,000 diagnosed prevalent cases of achondroplasia.
- The epidemiology model categorizes age-specific cases into nine groups: 0–4, 5–10, 11–15, 16–20, 21–30, 31–40, 41–50, 51–60, and over 60. DelveInsight’s estimates indicate that the 5–10 age group was the most affected in the US, with around 4,000 cases reported in 2023.
- Achondroplasia is estimated to occur in 1 out of every 15,000 to 40,000 live births, making it one of the most prevalent types of skeletal dysplasia.
- Key Achondroplasia Companies: BridgeBio/ QED Therapeutics , Ribomic Inc, Sanofi, Changchun GeneScience Pharma, Ascendis Pharma A/S, QED Therapeutics, Inc, BioMarin Pharmaceuticals, BioMarin Pharmaceutical, and others
- Key Achondroplasia Therapies: VOXZOGO (vosoritide), Infigratinib, RBM-007, SAR-442501, Recombinant human growth hormone, Infigratinib, TransCon CNP, Infigratinib, vosoritide, BMN 111, and others
- The Achondroplasia market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Achondroplasia pipeline products will significantly revolutionize the Achondroplasia market dynamics.
Achondroplasia Overview
Achondroplasia is a genetic disorder that causes abnormal bone growth, resulting in short stature and disproportionate limb size. It is the most common form of dwarfism, typically characterized by a larger head, short arms and legs, and a normal-sized trunk. The condition is caused by mutations in the FGFR3 gene, which affects the development of cartilage into bone. Individuals with achondroplasia may also experience other health issues such as joint problems, spinal abnormalities, and ear infections. Treatment focuses on managing symptoms, and while there is no cure, growth hormone therapy and other treatments can help improve quality of life.