The Hypophosphatasia (HPP) is a rare genetic disease caused by mutations in the ALPL gene. It is a metabolic disorder that effects multiple body systems and leads to life-threatening complications. This disease leads to abnormal development of the bones due to defective bone mineralization and can lead to rickets, tempering of the bones that result in different types of bone abnormalities. The sign and symptoms include soft bones, hypercalcemia, short stature and weak bones with loss of mobility, and may also result in premature death. According to U.S. Food and Drug Administration, it is estimated that in about 100,000 newborns, one suffers severe hypophosphatasia.
The mildest form of hypophosphatasia is called odontohypophosphatasia and is affects teeth. Infantile and perinatal hypophosphatasia is an autosomal recessive disease. Hypophosphatasia is an ultra-rare disease affecting < 20 people per million and it effects people of all ages as it is caused due to deficiency of an enzyme known as tissue non-specific alkaline phosphatase (TNSALP), which leads to high level of tissue non-specific alkaline phosphatase (TNSALP) substrates, including inorganic pyrophosphate. The adult form of this disease and odontohypophosphatasia is an autosomal recessive or autosomal dominant disease.
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Treatment and management of hypophosphatasia (HPP) is symptomatic and supportive, it aims at addressing symptoms of disease such as thiazide diuretics for hypercalcemia, ventilator support, occupational therapy and surgery for bone fractures. In October 2015, the enzyme replacement therapy has been recently approved for the treatment of abnormal bone structure in people with onset hypophosphatasia (HPP). The HPP community are helping to bring programs that will bring new and treatment options to people with rare diseases. Currently, clinical trials are conducted, wherein a large number of organization are actively participating in several research programs to develop novel therapies for infantile and juvenile-onset hypophosphatasia treatment.
Introduction of several novel therapy, breakthrough therapy designation, orphan drug designation for rare diseases, financial incentives and grants by the government, clinical studies on safety and efficacy of products, is expected to be the biggest hit in the global infantile and juvenile-onset hypophosphatasia treatment over the forecast period. Gaps in making hypophosphatasia (HPP) health priority, patients’ non-adherence to therapy regime, high therapy cost, less number of approved therapies, failure in clinical trial at the late phase are some of the factors restraining the infantile and juvenile-onset hypophosphatasia treatment market.
The global Infantile and Juvenile-onset Hypophosphatasia Treatment market is segmented on basis of treatment type, end user and geography
Segment by Treatment Type
Enzyme Replacement Therapy
Segment by End User
Clinical Research Organizations
Global Infantile and Juvenile-onset Hypophosphatasia Treatment market is segmented by treatment type, and end user. Based on the treatment type, global Infantile and Juvenile-onset Hypophosphatasia treatment market is segmented as supportive care and enzyme replacement therapy. On the basis of end user, the global infantile and juvenile-onset hypophosphatasia treatment market is segmented into hospital, clinics, clinical research organizations and others. Rise in patient population of hypophosphatasia, increased awareness and new product/therapy launch is said to drive the global Infantile and Juvenile-onset hypophosphatasia treatment market during the forecast period. Whereas inadequate access to health care, cost and availability of essential medicines and lack of standardized tools for diagnosis and treatment will hamper the growth of global infantile and juvenile-onset hypophosphatasia treatment market.
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By Geography, the global Infantile and juvenile-onset hypophosphatasia treatment market is segmented into five broad regions viz. North America, Latin America, Europe, Asia-Pacific, and the Middle East & Africa. North America is estimated to lead the global Infantile and Juvenile-onset Hypophosphatasia Treatment market due to rise in cases of hypophosphatasia in United States, Europe market is also projected to experience high growth in the near future. Asia Pacific market is anticipated to grow at the fastest growth rate during the forecast period, owing to factors such as rising standard of living, lifestyle changes, rise in research and development, government initiatives, drug designations such as breakthrough therapy, Orphan drug designation by FDA helps company to work collaboratively with sponsors to expedite the drug development process.
Some of the major players in global Infantile and Juvenile-onset Hypophosphatasia Treatment market are Alexion Pharmaceuticals Inc., Novartis A.G, and Others.