Asfotase alfa

Overview

Asfotase alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP). Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. By replacing deficient ALP, treatment with Asfotase Alfa aims to improve the elevated enzyme substrate levels and improve the body's ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. Asfotase alfa was first approved by Pharmaceuticals and Medicals Devices Agency of Japan (PMDA) on July 3, 2015, then approved by the European Medicine Agency (EMA) on August 28, 2015, and was approved by the U.S. Food and Drug Administration (FDA) on October 23, 2015. Asfotase Alfa is marketed under the brand name Strensiq® by Alexion Pharmaceuticals, Inc. The annual average price of Asfotase Alfa treatment is $285,000.

Background

Indication

Indicated for the treatment of patients with perinatal/infantile and juvenile onset hypophosphatasia (HPP).

Associated Conditions

Infantile-onset Hypophosphatasia
Juvenile-onset Hypophosphatasia
Perinatal-onset Hypophosphatasia

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa	2023/10/12	NCT06079372	Phase 3	Active, not recruiting	Alexion Pharmaceuticals, Inc.
A Prospective Sub-Study of the Global Hypophosphatasia Registry	2022/02/10	NCT05234567	N/A	Recruiting	Alexion Pharmaceuticals, Inc.
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)	2019/12/12	NCT04195763	N/A	Completed	Alexion Pharmaceuticals, Inc.
Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease	2019/12/06	NCT04189315	Phase 4	Withdrawn	Alexion Pharmaceuticals, Inc.
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)	2016/06/14	NCT02797821	Phase 2	Completed	Alexion Pharmaceuticals, Inc.
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)	2010/08/05	NCT01176266	Phase 2	Completed	Alexion Pharmaceuticals, Inc.
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)	2010/07/15	NCT01163149	Phase 2	Completed	Alexion Pharmaceuticals, Inc.

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
STRENSIQ	Alexion Pharmaceuticals, Inc.	25682-019	SUBCUTANEOUS	80 mg in 0.8 mL	3/14/2023
STRENSIQ	Alexion Pharmaceuticals, Inc.	25682-013	SUBCUTANEOUS	28 mg in 0.7 mL	3/14/2023
STRENSIQ	Alexion Pharmaceuticals, Inc.	25682-016	SUBCUTANEOUS	40 mg in 1 mL	3/14/2023
STRENSIQ	Alexion Pharmaceuticals, Inc.	25682-010	SUBCUTANEOUS	18 mg in 0.45 mL	3/14/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Strensiq	Alexion Europe SAS,103-105 rue Anatole France,92300 Levallois-Perret,France	Authorised	8/28/2015
Strensiq	Alexion Europe SAS,103-105 rue Anatole France,92300 Levallois-Perret,France	Authorised	8/28/2015
Strensiq	Alexion Europe SAS,103-105 rue Anatole France,92300 Levallois-Perret,France	Authorised	8/28/2015
Strensiq	Alexion Europe SAS,103-105 rue Anatole France,92300 Levallois-Perret,France	Authorised	8/28/2015

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
STRENSIQ SOLUTION FOR INJECTION 100 MG/ML	Alexion Pharma International Operations Limited (AAMF), Patheon Italia S.p.A	SIN17126P	INJECTION, SOLUTION	100mg/mL	10/29/2024
STRENSIQ SOLUTION FOR INJECTION 40 MG/ML	Alexion Pharma International Operations Limited (AAMF), Patheon Italia S.p.A	SIN17127P	INJECTION, SOLUTION	40mg/mL	10/29/2024

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
STRENSIQ asfotase alfa rch 18 mg/0.45 ml solution for injection vial	266985	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	1/14/2016
STRENSIQ asfotase alfa rch 28 mg/0.70 ml solution for injection vial	266986	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	1/14/2016
STRENSIQ asfotase alfa rch 12 mg/0.30 ml solution for injection vial	266984	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	1/14/2016
STRENSIQ asfotase alfa rch 40 mg/1.00 ml solution for injection vial	232546	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	1/14/2016
STRENSIQ asfotase alfa rch 80 mg/0.80 mL injection solution vial	232545	Alexion Pharmaceuticals Australasia Pty Ltd	Medicine	A	1/14/2016

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