The global skeletal dysplasia market size is expected to reach USD 3.6 billion by 2026, according to a new report by Grand View Research, Inc., registering a 4.0% CAGR over the forecast period. Increasing prevalence of this condition, especially in children, and growing adoption of orphan drugs are likely to contribute to the expansion of the market.
Among different disorders, achondroplasia is the most common condition. According to estimates published by the National Human Genome Research Institute in 2016, achondroplasia occurs in 1 in 15,000-40,000 live births. Although there are no current therapies approved specifically for achondroplasia, there are five therapies under clinical development.
Genetic defects attributable to the disorder include spontaneous mutations in the FGFR3 gene. Vosoritide by BioMarin targets inhibition of the FGFR3 pathway and presents a first-in-class drug for achondroplasia. The drug is in phase III clinical trial and is expected to enter the market in 2024. Clementia (Ipsen Group) and Regeneron are other market players targeting disorders such as multiple osteochondromas and fibrodysplasia ossificans progressiva (FOP). Approval of these pipeline therapies with orphan drug status during the forecast period will further support skeletal dysplasia market growth.
The medicines vary depending on the genetic cause of the disorder and only a handful of the disorders, such as XLH and hypophosphatasia, have approved curative therapies, including Ultragenyx Pharmaceuticals’ Crysvita and Alexion Pharmaceuticals’ Strensiq, respectively. Crysvita received FDA approval in 2018 whereas Strensiq received approval in 2015. The sales of Strensiq increased from USD 12.0 million in 2015 to USD 475.1 million in 2018. Higher uptake of these orphan drugs signal growth of the market.
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Further key findings from the report suggest:
Depending on the disorder, patients develop multiple symptoms ranging from spinal deformities to abnormal dental development. These disorders limit mobility and functional abilities in patients
Commonly occurring deformities in these disorders include abnormal skeletal development that require orthopedic procedures and other interventions to relieve movement obstructions
Early identification is crucial for timely medical management. The treatment for skeletal dysplasia is restricted largely to supportive care
Surgical therapy is the most effective treatment option for achondroplasia type segment of skeletal dysplasia market. There is currently no definitive treatment for FOP. However, a brief course of high-dose corticosteroids, such as Prednisone, may help reduce intense inflammation and tissue swelling seen in early stages.