Hypophosphatasia (HPP) is a rare genetic disorder characterized by the abnormal development of bones and teeth. These abnormalities occur due to defective mineralization such as calcium and phosphorus.
Hypophosphatasia Treatment: Introduction
- Hypophosphatasia (HPP) is a rare genetic disorder characterized by the abnormal development of bones and teeth. These abnormalities occur due to defective mineralization such as calcium and phosphorus. These minerals are required for proper hardness and strength. Defective mineralization results in bones becoming soft and more prone to fracture.
- Hypophosphatasia symptoms can vary greatly from one person to another, sometimes even among members of the same family. HPP is caused by mutations in the tissue nonspecific alkaline phosphatase (ALPL) gene, also known as TNSALP gene. Such mutations lead to low activity of the tissue nonspecific alkaline phosphatase enzyme. Depending on the specific form, HPP can be inherited in an autosomal recessive or autosomal dominant manner.
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Key Drivers of Global Hypophosphatasia Treatment Market
- Increasing number of patients with hypophosphatasia, family history of hypophosphatasia, strong product pipeline, and increasing financial support to researchers for developing new innovative drugs for treatment of hypophosphatasia are likely to drive the hypophosphatasia market during the forecast period. In addition, increase in special designation from the regulatory authorities to offer a treatment to the patients as quickly as possible is enhancing the market.
- According to the National organization for Rare Disorders, the overall incidence and prevalence of hypophosphatasia is unknown, but in Canada, the severe form of hypophosphatasia affects approximately 1 in 100,000 live births.
- In other hand, patent expiration of major brands and less treatment options available are likely to restrain the growth of this market
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Odontohypophosphatasia Segment Expected to be Most Promising Segment
- Based on type, the global hypophosphatasia treatment market can be segmented into odontohypophosphatasia, pseudohypophosphatasia, and others
- The odontohypophosphatasia segment accounted for the highest share of the hypophosphatasia treatment market in 2019. This segment is expected to dominate the hypophosphatasia treatment market due to high number of patients with odontohypophosphatasia, which is characterized by loss of premature teeth in childhood or loss of teeth in adulthood.
Medication Segment to Witness Significant Growth
- In terms of treatment type, the global hypophosphatasia treatment market can be divided into medication and surgery. The medication segment is further divided into asfotase alfa, non-steroidal anti-inflammatory drugs, and others.
- The medication segment generated the highest revenue in 2019 and is anticipated to witness growth during the forecast period. This can be attributed to launch of products for treatment of hypophosphatasia from major manufacturers. In 2015, the U.S. Food and Drug Administration (FDA) approved Strensiq (asfotase alfa) as the first approved medical treatment for infantile, perinatal, and juvenile-onset HPP.
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Hospitals Pharmacies Segment to be Most Promising Segment
- In terms of distribution channel, the global hypophosphatasia treatment market can be divided into hospital pharmacies, retail pharmacies, and online sales. The hospital pharmacies segment generated the highest revenue in 2019 and is anticipated to witness growth during the forecast period due to most of major products are prescription products, which is available in hospital pharmacies.
North America to Dominate Global Hypophosphatasia Treatment Market
- In terms of region, the global hypophosphatasia treatment market can be categorized into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global hypophosphatasia treatment market in 2019 and is expected to maintain its leading position during the forecast period. This dominance of the region can be attributed to increasing research & development activity from major market players, increase in product offerings, greater affordability, and rise in demand for early treatment.
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- The market in Asia Pacific is projected to register highest growth during the forecast period, owing to increase in adoption of new products and rise in demand for hypophosphatasia treatment in emerging countries such as India and China
Key Manufacturers Operating in Global Market
The global hypophosphatasia treatment market was highly fragmented in 2019. Key manufacturers operating in the global market are:
- Kirin Holdings Company
- Vericel Corporation
- Mereo BioPharma Group plc
- Novartis AG
- AM?Pharma B.V.
- Alexion
- Bayer AG
- Pfizer, Inc.
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