Adolescent

For adolescents, STRENSIQ strengthens bones1,2

See below for the results of the 6-minute walk test (6MWT) after treatment with STRENSIQ.

All patient images are hypothetical.
Adolescent with headphones and backpack
Bone mineralization Radiographic evidence 6-minute walk test
INFANT/YOUNG CHILD INFANT/YOUNG CHILD PEDIATRICADOLESCENT ADOLESCENTADULT
Study 11-3ENB-002-08/
ENB-003-08		 Study 21,5ENB-010-10 Study 31,5,6ENB-006-09/
ENB-008-10		 Study 47ENB-009-10
Design Prospective, single-arm trial and extension in patients with severe perinatal/infantile-onset HPP Prospective, open-label trial in patients with perinatal/infantile-onset HPP Prospective, open-label trial and extension in patients with juvenile- or perinatal/infantile-onset HPP Multicenter, randomized, open-label concurrent control trial including adolescents and adults with pediatric-onset HPP
Duration 7 years 6 years 7 years 5 years
Primary treatment phase 6 months 1-6 years 6 months 6 months
Extension 6.5 years 1-6 years 6.5 years 4.5 years
Number of patients 11 69 13(9 reached adolescencea
during the study8)		 19(13 adults,b
6 adolescents)a
Age at inclusion ≤3 years ≤5 years 6 to 12 years 13 to 65 years

Study 11-3ENB-002-08/ENB-003-08

INFANT/YOUNG CHILD
DESIGN

Prospective, single-arm trial and extension in patients with severe perinatal/infantile-onset HPP

DURATION

7 years

PRIMARY Tx PHASE

6 months

EXTENSION

6.5 years

# OF PATIENTS

11

AGE AT INCLUSION

≤3 years

Study 21,5ENB-010-10

INFANT/YOUNG CHILD
DESIGN

Prospective, open-label trial in patients with perinatal/infantile-onset HPP

DURATION

6 years

PRIMARY Tx PHASE

1-6 years

EXTENSION

—

# OF PATIENTS

69

AGE AT INCLUSION

≤5 years

Study 31,5,61,2,61,4,6ENB-006-09/ENB-008-10

PEDIATRICADOLESCENT
DESIGN

Prospective, open-label trial and extension in patients with juvenile- or perinatal/infantile-onset HPPa

DURATION

7 years

PRIMARY Tx PHASE

6 months

EXTENSION

6.5 years

# OF PATIENTS

13

(9 reached adolescenceab
during the study382)
AGE AT INCLUSION

6 to 12 years

Study 417ENB-009-10

ADOLESCENTADULT
DESIGN

Multicenter, randomized, open-label concurrent control trial including adolescents and adults with pediatric-onset HPPa

DURATION

5 years

PRIMARY Tx PHASE

6 months

EXTENSION

4.5 years

# OF PATIENTS

19

(13 adults,b
6 adolescents)a
AGE AT INCLUSION

13 to 65 years

Abbreviation: HPP, hypophosphatasia.

aAdolescents in the study were aged 13 to 17 years.6-8

bAdults in the study were aged ≥18 years and 12/13 had pediatric-onset HPP.7

aAdolescents in the study were aged 13 to 17 years.2,3

aThe data represented are from subgroup analysis.2

bAdolescents in the study were aged 13 to 17 years.1,2,81,2

aData represent subgroup analysis of adults with pediatric-onset HPP.

bAdults in the study were aged ≥18 years and 12/13 had pediatric-onset HPP.1

Bone mineralization (primary endpoint)

With STRENSIQ, most adolescent patients achieved complete healing of hypophosphatasia (HPP)-related rickets2

  • After 6 months, 67% (6/9) of patients experienced complete or substantial healing of HPP-related rickets as measured by Radiographic Global Impression of Change (RGI-C); by 7 years, the responder rate was 100% (9/9)2
    • The RGI-C scale quantifies change over time in mineralization and bone structure3
  • Efficacy results reflect data for 9 individuals who reached adolescence (aged 13 to 17 years) during the course of the study2
Efficacy results for bone mineralization with Strensiq®
Efficacy results for bone mineralization with Strensiq®

Radiographic evidence (primary endpoint)

STRENSIQ strengthens bones1,2

KNEE

Substantial healing of HPP-related rickets1,2
12 YEARS OF AGE (AT BASELINE)4
Knee 12 years
17 YEARS OF AGE (AT 5 YEARS)4
Knee 17 years
After 5 years of STRENSIQ treatment, patient 1 had an RGI-C score of +2.67.

6-minute walk test (secondary endpoint)

STRENSIQ normalizes mobility as evidenced by 6MWT results in adolescent patients through 7 years1,2

Significant improvement in 6MWT was seen as early as 3 months and was sustained at all time points measured through 7 years.2

Measurement of mobility with the 6-minute walk test for adolescents
Measurement of mobility with the 6-minute walk test for adolescents
  • At 6 months, there was a 36% median change from baseline in distance walked (511 meters vs 350 meters) (P=0.0078)2
  • At 7 years, there was a 68% median change from baseline in distance walked (593 meters vs 350 meters) (P=0.0078)2
    • For adolescent patients with HPP, the minimal clinically important difference (MCID) is estimated at 33 meters and 43 meters based on 2 distribution-based methods5

STRENSIQ may help reduce or reverse the natural history of HPP progression as seen by improved bone mineralization and walking distance.1,2

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Important Safety Information and Indication for STRENSIQ® (asfotase alfa) 40mg/mL vial
Important Safety Information
WARNINGS AND PRECAUTIONS
  • Hypersensitivity Reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and have been observed more than 1 year after treatment initiation. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia.
    Inform patients and/or caregivers of the signs and symptoms of hypersensitivity reactions and have them seek immediate medical care should signs and symptoms occur. If a severe hypersensitivity reaction occurs, discontinue STRENSIQ treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.
  • Lipodystrophy: Localized lipodystrophy, including lipoatrophy (depression in the skin) and lipohypertrophy (enlargement or thickening of tissue), has been reported at injection sites after several months in patients treated with STRENSIQ in clinical trials. Advise patients to follow proper injection technique and to rotate injection sites.
  • Ectopic Calcifications: Patients with HPP are at increased risk for developing ectopic calcifications. Events of ectopic calcification, including ophthalmic (conjunctival and corneal) and renal (nephrocalcinosis, nephrolithiasis), have been reported in the clinical trial experience with STRENSIQ. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported resulting from the occurrence of ectopic calcifications.
    Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function.
  • Possible Immune-Mediated Clinical Effects: In clinical trials, most STRENSIQ-treated patients developed anti-asfotase alfa antibodies and neutralizing antibodies which resulted in reduced systemic exposure of asfotase alfa. In postmarketing reports, some STRENSIQ-treated patients with initial therapeutic response subsequently developed recurrence and worsening in disease-associated laboratory and radiographic biomarkers (some in association with neutralizing antibodies) suggesting possible immune-mediated effects on STRENSIQ’s pharmacologic action resulting in disease progression. The effect of anti-asfotase alfa antibody formation on the long-term efficacy of STRENSIQ is unknown. There are no marketed anti-asfotase alfa antibody tests. If patients experience progression of HPP symptoms or worsening of disease-associated laboratory and imaging biomarkers after a period of initial therapeutic response to STRENSIQ, consider obtaining anti-asfotase alfa antibody testing by contacting STRENSIQ Medical Information at Alexion at 1-888-765-4747 or by email at medinfo@alexion.com. Close clinical follow up is recommended.
ADVERSE REACTIONS
  • Overall, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%). Other common adverse reactions included lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ.
DRUG INTERACTIONS
    Drug Interference with Laboratory Tests:
  • Laboratory tests utilizing alkaline phosphatase (ALP) as a detection reagent could result in erroneous test results for patients receiving treatment due to the presence of asfotase alfa in clinical laboratory samples. Inform laboratory personnel that the patient is being treated with STRENSIQ and discuss use of an alternative testing platform which does not utilize an ALP-conjugated test system.
  • Elevated serum ALP measurements detected through clinical laboratory testing are expected in patients receiving STRENSIQ due to circulating concentrations of asfotase alfa and may be unreliable for clinical decision making.
SPECIAL POPULATIONS
  • Pregnancy & Lactation: There are no available data on STRENSIQ use in pregnant women, the presence of STRENSIQ in human milk, or the effects on the breastfed infant or on milk production, to inform a drug associated risk.
Indication

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

Please see STRENSIQ (asfotase alfa) full Prescribing Information.
Important Safety Information and Indication for STRENSIQ® (asfotase alfa) 40mg/mL vial
Important Safety Information
WARNINGS AND PRECAUTIONS
  • Hypersensitivity Reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and have been observed more than 1 year after treatment initiation. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia.
    Inform patients and/or caregivers of the signs and symptoms of hypersensitivity reactions and have them seek immediate medical care should signs and symptoms occur. If a severe hypersensitivity reaction occurs, discontinue STRENSIQ treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.
  • Lipodystrophy: Localized lipodystrophy, including lipoatrophy (depression in the skin) and lipohypertrophy (enlargement or thickening of tissue), has been reported at injection sites after several months in patients treated with STRENSIQ in clinical trials. Advise patients to follow proper injection technique and to rotate injection sites.
  • Ectopic Calcifications: Patients with HPP are at increased risk for developing ectopic calcifications. Events of ectopic calcification, including ophthalmic (conjunctival and corneal) and renal (nephrocalcinosis, nephrolithiasis), have been reported in the clinical trial experience with STRENSIQ. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported resulting from the occurrence of ectopic calcifications.
    Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function.
  • Possible Immune-Mediated Clinical Effects: In clinical trials, most STRENSIQ-treated patients developed anti-asfotase alfa antibodies and neutralizing antibodies which resulted in reduced systemic exposure of asfotase alfa. In postmarketing reports, some STRENSIQ-treated patients with initial therapeutic response subsequently developed recurrence and worsening in disease-associated laboratory and radiographic biomarkers (some in association with neutralizing antibodies) suggesting possible immune-mediated effects on STRENSIQ’s pharmacologic action resulting in disease progression. The effect of anti-asfotase alfa antibody formation on the long-term efficacy of STRENSIQ is unknown. There are no marketed anti-asfotase alfa antibody tests. If patients experience progression of HPP symptoms or worsening of disease-associated laboratory and imaging biomarkers after a period of initial therapeutic response to STRENSIQ, consider obtaining anti-asfotase alfa antibody testing by contacting STRENSIQ Medical Information at Alexion at 1-888-765-4747 or by email at medinfo@alexion.com. Close clinical follow up is recommended.
ADVERSE REACTIONS
  • Overall, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%). Other common adverse reactions included lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ.
DRUG INTERACTIONS
    Drug Interference with Laboratory Tests:
  • Laboratory tests utilizing alkaline phosphatase (ALP) as a detection reagent could result in erroneous test results for patients receiving treatment due to the presence of asfotase alfa in clinical laboratory samples. Inform laboratory personnel that the patient is being treated with STRENSIQ and discuss use of an alternative testing platform which does not utilize an ALP-conjugated test system.
  • Elevated serum ALP measurements detected through clinical laboratory testing are expected in patients receiving STRENSIQ due to circulating concentrations of asfotase alfa and may be unreliable for clinical decision making.
SPECIAL POPULATIONS
  • Pregnancy & Lactation: There are no available data on STRENSIQ use in pregnant women, the presence of STRENSIQ in human milk, or the effects on the breastfed infant or on milk production, to inform a drug associated risk.
Indication

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

Please see STRENSIQ (asfotase alfa) full Prescribing Information.
; ;

References

1. Whyte MP, Madson KL, Phillips D, et al. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016;1(9):e85971.

2. Data on file. Alexion Pharmaceuticals.

3. Whyte MP, Fujita KP, Moseley S, Thompson D, McAlister W. Validation of a novel scoring system for changes in skeletal manifestations of hypophosphatasia in newborns, infants, and children: the Radiographic Global Impression of Change Scale. J Bone Miner Res. 2018;33(5):868-874.

4. Whyte MP, Madson KL, Phillips D, et al. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016;1(9)(suppl):e85971.

5. Phillips D, Tomazos IC, Moseley S, L’Italien G, Gomes da Silva H, Lerma Lara S. Reliability and validity of the 6-minute walk test in hypophosphatasia. JBMR Plus. 2019;3(6):e10131.

6. STRENSIQ [package insert]. Alexion Pharmaceuticals Inc.

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