Exonucleotide pyrophosphatase / phosphodiesterase 1 (ENPP1) inhibitors for treatment of hypophosphoesterase disorders
By administering ENPP1 inhibitors to suppress PPi production, the bone and muscle pain problems associated with hypophospholipase syndrome are resolved, bone mineralization is promoted, periodontal health is improved, and an effective treatment and prevention method is provided.
Patent Information
- Application Number
- CN202480032171.4
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Priority Date
- 2023-04-13
- Filing Date
- 2024-03-29
- Publication Date
- 2026-01-09
AI Technical Summary
Hypophosphatase syndrome (HPP) is a rare, inherited metabolic disorder that causes the accumulation of ethanolamine phosphate, inorganic pyrophosphate, and 5-pyridoxal phosphate in the blood or urine, leading to severe symptoms such as bone mineralization defects, fractures, muscle pain, and periodontal disease. There is currently no effective treatment available.
By administering an extracellular nucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) inhibitor, PPi production is suppressed, extracellular PPi levels are restored, and HPP symptoms are relieved.
It significantly relieves bone and muscle pain symptoms in HPP, promotes bone mineralization, reduces the risk of fractures, improves periodontal health, and provides effective treatment and prevention.