Substance / Medication

Asfotase alfa

Overview

Active Ingredient
asfotase alfa
RxNorm CUI
1720259

Indications

® STRENSIQis indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

Labeler: Alexion Pharmaceuticals, Inc.Updated: 2025-12-12T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Warnings and Precautions (5.1) [see] Initiate

Contraindications

When this intervention should not be used

None.

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 DailyMed

Monitoring & 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.

Clinical Trials

1 trials linked to this intervention

1
Total Trials
0
Recruiting
1
With Results

Research Evidence

Published studies and systematic reviews

Sort:
Efficacy and safety of asfotase alfa in patients with hypophosphatasia: A systematic review.
Shirinezhad Amirhossein, Esmaeili Sina, Azarboo Alireza et al. · Bone · 2024
PMID: 39089608Meta-Analysis
Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa.
Gospe Sidney M, Santiago-Turla Cecilia, DeArmey Stephanie M et al. · Cornea · 2019
PMID: 30969260RCT
Pharmacodynamics of asfotase alfa in adults with pediatric-onset hypophosphatasia.
Seefried Lothar, Kishnani Priya S, Moseley Scott et al. · Bone · 2021
PMID: 32987199Trial
Pharmacokinetics of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia.
Pan Wei-Jian, Pradhan Rajendra, Pelto Ryan et al. · J Clin Pharmacol · 2021
PMID: 33822385TrialFull text (PMC)
Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study.
Hofmann Christine E, Harmatz Paul, Vockley Jerry et al. · J Clin Endocrinol Metab · 2019
PMID: 30811537TrialFull text (PMC)
Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial.
Whyte Michael P, Simmons Jill H, Moseley Scott et al. · Lancet Diabetes Endocrinol · 2019
PMID: 30558909Trial
Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia.
Kishnani Priya S, Rockman-Greenberg Cheryl, Rauch Frank et al. · Bone · 2019
PMID: 30576866Trial
Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial.
Kitaoka Taichi, Tajima Toshihiro, Nagasaki Keisuke et al. · Clin Endocrinol (Oxf) · 2017
PMID: 28374482Trial
Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia.
Whyte Michael P, Rockman-Greenberg Cheryl, Ozono Keiichi et al. · J Clin Endocrinol Metab · 2016
PMID: 26529632TrialFull text (PMC)

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 DailyMed

Related 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.

Medical Disclaimer

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
Asfotase alfa (substance)
SNOMED CT
715255009
UMLS CUI
C3490795
RxNorm CUI
1720259
Labeler
Alexion Pharmaceuticals, Inc.

Clinical Data

This intervention maps to 2 entities in the Ltrl knowledge graph.

2
Conditions
0
Biomarkers
0
Specialists
0
Symptoms
1
Clinical Trials

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.