Skeletal dysplasia market encompasses a broad number of genetic and rare disorders that affect mainly the cartilage and bone. The report provides segmentation of the market by disorder type into the following: X-linked Hypophosphatemia (XLH), hypophosphatasia, achondroplasia, multiple osteochondromas, and fibrodysplasia Ossificans Progressiva (FOP). Depending on the disorder, patients develop multiple symptoms ranging from spinal deformities to abnormal dental development. As these disorders limit mobility and functional abilities in patients, early identification is crucial for timely medical management.
The skeletal dysplasia market by treatment is restricted largely to supportive care and segmented into medication and surgeries. Commonly occurring deformities in these disorders include abnormal skeletal development that require orthopedic procedures and other interventions to relieve movement obstructions. For instance, osteotomies are performed for bowing of lower limbs & forearms that occurs in nearly all disorders. Endodontic procedures and craniectomies are some of the other procedures carried out to treat developmental abnormalities.
The market by medication includes multiple drug classes that provide mostly symptomatic care. Broadly, these include growth hormones, parathyroid hormones, mast cell inhibitors, and muscle relaxants, among others. 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 is a recent entrant that registered 85 million USD in sales, in 2018. Whereas Strensiq’s sales increased from 12 million USD in 2015 to nearly 350 million USD in 2017. Higher uptake of these orphan drugs signal growth of the market.
Among different disorders, achondroplasia is the most common condition with a prevalence rate of 5 in 100000 and incidence rate of 1 in 20,000 live births. Although there are no current therapies approved specifically for achondroplasia, there are five therapies under clinical development. Genetic defects attributable for the disorder are spontaneous mutations in the FGFR3 gene. Vosoritide by BioMarin Pharmaceuticals targets inhibition of the FGFR3 pathway and presents a first-in-class drug for achondroplasia. The drug is in phase III and expected to enter the market in 2024. Clementia Pharmaceuticals and Regeneron Pharmaceuticals are the other market players targeting disorders such as multiple osteochondromas and FOP. Approval of these pipeline therapies with orphan drug status during the forecast period will further support market growth.
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