amicus-therapeutics · Example Payload

Amicus Pombiliti Medicine Example

Example Amicus medicine record for Pombiliti (cipaglucosidase alfa-atga), the enzyme replacement component of the two-component Pombiliti + Opfolda regimen for adults with late-onset Pompe disease.

BiotechnologyPharmaceuticalsRare DiseasesGenetic DiseasesFabry DiseasePompe DiseaseLysosomal Storage DisordersEnzyme Replacement TherapyPharmacological ChaperonesClinical ResearchLife SciencesPatient Advocacy

Amicus Pombiliti Medicine Example is an example object payload from amicus-therapeutics, with 2 top-level fields. It illustrates the shape of data this provider's APIs accept or return.

Top-level fields

descriptionmedicine

Example Payload

{
  "description": "Example Amicus medicine record for Pombiliti (cipaglucosidase alfa-atga), the enzyme replacement component of the two-component Pombiliti + Opfolda regimen for adults with late-onset Pompe disease.",
  "medicine": {
    "medicineId": "amx-med-pombiliti",
    "brandName": "Pombiliti",
    "genericName": "cipaglucosidase alfa-atga",
    "drugClass": "Enzyme Replacement Therapy",
    "routeOfAdministration": "Intravenous",
    "indication": "Late-onset Pompe disease",
    "patientPopulation": "Adults with late-onset Pompe disease (LOPD) weighing 88 lbs or more who are not improving on their current enzyme replacement therapy",
    "combinationWith": ["Opfolda"],
    "mechanismOfAction": "Bis-M6P-enriched ERT designed to deliver active GAA enzyme directly to muscle cells for improved binding and effective uptake",
    "approvals": [],
    "productSite": "https://www.pombilitiopfolda.com"
  }
}