Overview
Indications
POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
Boxed Warning
FDA Black Box Warning
Hypersensitivity Reactions Including Anaphylaxis Warnings and Precautions (5.1) [see]. Patients treated with POMBILITI have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should
Contraindications
When this intervention should not be used
Warnings and Precautions (5.4) Use in Specific Populations (8.1) [seeand]. POMBILITI in combination with Opfolda is contraindicated in pregnancy
Indications & Related Conditions
Conditions associated via SNOMED clinical relationships
Administration & Protocol
Dosing, route, and treatment protocol
Detailed dosage and administration information is available in the full FDA drug label.
View full prescribing information on DailyMedMonitoring & Follow-Up
Biomarkers relevant to this intervention via related conditions
No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.
Research Evidence
Published studies and systematic reviews
Research data from MEDLINE/PubMed
Benefits & Expected Outcomes
Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.
Risks & Side Effects
Adverse reaction and safety data for this drug is sourced from the FDA-approved label.
View adverse reactions & drug interactions on DailyMedRelated Symptoms
Symptoms associated with conditions this intervention addresses
No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.
Alternatives & Comparisons
Alternative treatments, comparison data, and clinical decision support are pending physician authorship.
This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.
Do not start, stop, or change any treatment without consulting your healthcare provider.
Quick Facts
- Type
- Substance / Medication
- Fully Specified Name
- Cipaglucosidase alfa (substance)
- SNOMED CT
- 1025331000122100
- UMLS CUI
- C5421138
- RxNorm CUI
- 2667433
- Labeler
- AMICUS THERAPEUTICS US, LLC
Clinical Data
This intervention maps to 1 entities in the Ltrl knowledge graph.
Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.