Asfotase alfa is an enzyme replacement therapy used for the treatment of perinatal/infantile and juvenile onset hypophosphatasia (HPP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth.
- Mechanism
- Curator reviewed
HPP is caused by a deficiency in TNSALP (tissue non-specific alkaline phosphatase) enzyme activity, which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth which inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia (softening of bones) once growth plates close, along with muscle weakness. Replacement of the TNSALP enzyme upon Asfotase alfa treatment reduces the enzyme substrate levels.
- Primary indication
- Indicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).Curator reviewed · 3 structured indications
- Formula / weight
- C7108H11008N1968O2206S56 · 180000.0 Da (Approximate)
- First approval
- Canada, 2015 · United States, 2015 · European Union, 2016
- Code names
- ALXN-1215 · ENB-0040 · sALP-FcD10
- Brand names
Resolves to
What you can answer from here — as of June 03, 2022
Which other approved drugs treat the same conditions as Asfotase alfa, and which companies have late-stage candidates in those indications?
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